Major histocompatibility complex (MHC) class I molecules are transmembrane glycoproteins expressed on the surface of nearly all nucleated cells in vertebrates, functioning to display short peptides derived from intracellular proteins to cytotoxic CD8+ T cells as part of the adaptive immune response.[1] These molecules enable the immune system to distinguish healthy cells from those infected by viruses, transformed by cancer, or otherwise compromised, thereby triggering targeted destruction of aberrant cells while inhibiting natural killer (NK) cells through recognition of self-peptides.[2] Structurally, MHC class I consists of a polymorphic α heavy chain non-covalently associated with the invariant β2-microglobulin (β2m) light chain; the α chain features three extracellular domains (α1, α2, and α3), where the α1 and α2 domains form a peptide-binding groove that accommodates peptides typically 8–10 amino acids in length, anchored by specific residues in polymorphic pockets.[1][2]The biosynthesis and antigen presentation pathway of MHC class I molecules occur primarily in the endoplasmic reticulum (ER), where newly synthesized heavy chains associate with chaperones like calnexin and calreticulin before β2m binding; peptides generated in the cytosol by the proteasome are transported into the ER via the transporter associated with antigen processing (TAP), trimmed by endoplasmic reticulum aminopeptidases (ERAPs), and loaded onto MHC class I with assistance from the peptide-loading complex, including tapasin.[2] This process ensures stable peptide-MHC complexes are transported to the cell surface via the Golgi apparatus for immune surveillance.[1] Genetically, the genes encoding human MHC class I (known as human leukocyte antigens or HLA-A, HLA-B, and HLA-C) are located in the MHC region on the short arm of chromosome 6, exhibiting extreme polymorphism with over 29,000 alleles identified (as of September 2025), which enhances population-level immune diversity by allowing presentation of a broad repertoire of peptides; inheritance is codominant and follows Mendelian patterns.[1][2][3]Evolutionarily, MHC class I molecules emerged around 500 million years ago, with their polymorphism maintained through balancing selection, including pathogen-driven pressures and mate choice preferences.[2] In addition to classical MHC class I, non-classical variants like HLA-E, HLA-F, and HLA-G play specialized roles, such as modulating NK cell activity or contributing to immune tolerance during pregnancy.[2] While ubiquitous in expression, levels of MHC class I can be downregulated by certain viruses or tumors to evade detection, underscoring their central role in immune homeostasis.[1]
Structure
Molecular Components
The major histocompatibility complex (MHC) class I molecule is composed of a polymorphic heavy chain, also known as the alpha chain (α chain), which is a glycoprotein with an approximate molecular weight of 45 kDa.[4] This heavy chain consists of three extracellular domains—α1, α2, and α3—a transmembrane region that anchors it to the cell membrane, and a short cytoplasmic tail.[4] In humans, the heavy chain is encoded by one of the highly polymorphic genes HLA-A, HLA-B, or HLA-C, located within the MHC region on chromosome 6.[4]Non-covalently associated with the heavy chain is β2-microglobulin (β2m), a non-polymorphic light chain with a molecular weight of approximately 12 kDa that is essential for the structural stability of the MHC class I complex.[5] The β2m subunit is encoded by the separate B2M gene on chromosome 15 and shares structural similarity with the immunoglobulin domain, particularly resembling the α3 domain of the heavy chain.The heavy chain undergoes post-translational modifications, including N-linked glycosylation primarily at a conserved asparagine residue (Asn86) in the α1 domain, which contributes to protein folding, quality control, and trafficking within the ER.[6] This glycosylation site is present across different HLA alleles, though the extent of glycan processing can vary.[7]
Architecture and Domains
The major histocompatibility complex (MHC) class I molecule forms a heterodimeric complex composed of a polymorphic heavy chain, also known as the α chain, and the invariant light chain β₂-microglobulin (β₂m). The heavy chain consists of three extracellular domains—α₁, α₂, and α₃—linked to a transmembrane helix and a short cytoplasmic tail, while β₂m associates non-covalently with the extracellular portion. The α₁ and α₂ domains, each comprising approximately 90 amino acids, fold into a β-sheet platform topped by α-helices that create a cleft for peptide binding, whereas the membrane-proximal α₃ domain adopts an immunoglobulin-like fold essential for interaction with the CD8 co-receptor on cytotoxic T cells.[8][9]The structural integrity of this platform relies on intimate interactions between the domains and β₂m. β₂m binds to the underside of the α₁-α₂ platform, stabilizing the heavy chain through extensive hydrogen bonding networks involving conserved residues, such as those in the β-strands of β₂m with complementary regions in α₁ and α₂. Additionally, an intra-domain disulfide bridge (Cys101-Cys164) within the α₂ domain, along with inter-domain hydrogen bonds, maintains the helical architecture of the peptide-binding cleft, preventing unfolding in the absence of peptide. These interactions ensure the molecule's stability on the cell surface, with β₂m playing a critical role in folding and assembly.[8][10]The three-dimensional structure of MHC class I was first elucidated in 1987 through X-ray crystallography of HLA-A2 at 3.5 Å resolution by Bjorkman et al., revealing a closed-ended groove formed by the α₁-α₂ helices, ideally suited for accommodating peptides of 8-10 residues in an extended conformation. This landmark study demonstrated how the platform elevates the peptide for T cellrecognition, with the α₃ domain positioned below to facilitate co-receptor engagement. Subsequent higher-resolution structures have refined these insights, confirming the conserved topology across human leukocyte antigen (HLA) and mouse H-2 alleles.[8]MHC class I molecules exhibit conformational flexibility, adopting open or closed states influenced by peptide occupancy. In the peptide-free or low-affinity state, the α-helices of the cleft partially separate, widening the groove to facilitate peptide entry, as observed in crystal structures of empty HLA-A*02:01. High-affinity peptide binding induces a closed conformation, where the helices converge, locking the peptide via hydrogen bonds and van der Waals interactions at the termini, thereby enhancing surface stability and immune surveillance. These dynamic transitions underscore the molecule's role in antigen presentation efficiency.[11][12]
Peptide Binding Groove
The peptide-binding groove of MHC class I molecules is a key structural feature located at the interface of the α1 and α2 domains, formed by two parallel α-helices that flank a floor composed of an eight-stranded antiparallel β-sheet.[8] This architecture creates a cleft approximately 25 Å long and 12 Å wide, designed to bind antigenic peptides derived from intracellular proteins. The groove's closed ends, enforced by conserved residues, restrict peptide length to typically 8-10 amino acids, ensuring stable presentation on the cell surface.[8][13]Within the groove, peptides are anchored primarily through interactions with specific pockets that accommodate side chains at defined positions. The A pocket at the N-terminal end binds the peptide's amino group via conserved hydrogen bonds, while the F pocket at the C-terminal end interacts with the carboxyl group and a hydrophobic anchor residue.[14] For example, in HLA-A*02:01, the F pocket favors a C-terminal leucine or valine, contributing to its preference for nonamer peptides with motifs like L/M at position 2 and V/L at the C-terminus.[15] Additional pockets (B through E) accommodate secondary anchors and variable residues, allowing peptide bulging for lengths up to 11-12 amino acids without disrupting overall binding.[14]Polymorphisms in MHC class I alleles primarily cluster in these pockets, altering binding specificity and the repertoire of presented peptides. Variations in pocket depth and residue composition can enhance or restrict anchor preferences; for instance, HLA-B27's deep B pocket, lined by aspartic acid at position 77 and other residues, selectively binds peptides with arginine at position 2, which is linked to its role in spondyloarthropathies.[16] Such allelic differences ensure diverse immune surveillance across populations by modulating peptide selectivity without compromising groove stability.[17]Peptides adopt an extended, polyproline II-like conformation within the groove, aligning parallel to the β-sheet floor in a manner resembling an additional antiparallel strand.[18] This is stabilized by a network of invariant hydrogen bonds from conserved tyrosine residues—such as Tyr7 in the β-sheet, Tyr59 in the α1 helix, Tyr159 in the α2 helix, and Tyr171—to the peptide's main-chain atoms, particularly at positions 1, 2, the C-terminus, and penultimate residue.[18] For peptides longer than nine residues, central bulges allow accommodation while maintaining anchor contacts, preserving the overall structural integrity essential for T cell recognition.[19]
Biosynthesis and Assembly
Intracellular Synthesis
MHC class I genes exhibit constitutive expression in nearly all nucleated cells, driven by conserved promoter elements including enhancer A, which binds NF-κB, and the interferon-stimulated response element (ISRE), which interacts with interferon regulatory factor (IRF) family members.[20] This basal transcription is further modulated by the SXY module, forming an enhanceosome with transcription factors such as RFX, CREB/ATF, and NF-Y to maintain steady-state levels essential for immune surveillance.[20] Inducible expression is primarily triggered by interferon-gamma (IFN-γ), which activates the JAK/STAT pathway, leading to STAT1 phosphorylation and subsequent induction of IRF1; these factors bind to the ISRE and gamma-activated site (GAS) elements, significantly upregulating MHC class I transcription during immune responses.[20][21]The heavy chain and β2-microglobulin (β2m), a non-covalently associated light chain essential for MHC class I stability, are both synthesized on free ribosomes in the cytosol.[22] The heavy chain, encoded by HLA-A, -B, or -C genes, features an N-terminal signal peptide that directs its co-translational translocation into the endoplasmic reticulum (ER) lumen via the Sec61 translocon, where the signal peptide is cleaved to initiate membrane integration.[22] β2m also features an N-terminal signal peptide that directs its co-translational translocation into the ER lumen via the Sec61 translocon, where the signal peptide is cleaved, allowing it to associate with the heavy chain during subsequent assembly steps.[22][5]Upon entry into the ER, the nascent heavy chain undergoes initial folding, beginning with binding to the lectin chaperone calnexin, which recognizes the monoglucosylated N-linked glycan on the α3 domain to facilitate quality control and prevent aggregation.[22] Disulfide bond formation in the α1 and α2 domains, critical for the peptide-binding groove structure, is catalyzed by the oxidoreductases ERp57 and protein disulfide isomerase (PDI), often in complex with calnexin or calreticulin.[22] Unfolded or misfolded nascent heavy chains are subject to rapid ER-associated degradation (ERAD), with a half-life of approximately 30-60 minutes, ensuring efficient turnover and preventing accumulation of defective molecules.[23]
Translocation to ER
Cytosolic proteins are primarily degraded by the 26S proteasome into short peptides, typically ranging from 8 to 11 amino acids in length, which serve as precursors for MHC class I antigen presentation. This degradation process generates a diverse pool of peptides from ubiquitinated proteins, with the proteasome's catalytic core, the 20S particle, cleaving internal bonds to produce these fragments. Under inflammatory conditions, interferon-gamma (IFN-γ) induces the formation of immunoproteasomes by incorporating specialized subunits such as LMP2 (β1i) and LMP7 (β5i), which alter the cleavage specificity to favor the production of peptides suitable for MHC class I binding.[24]The transporter associated with antigen processing (TAP), a member of the ATP-binding cassette (ABC) transporter family, facilitates the translocation of these cytosolic peptides into the endoplasmic reticulum (ER) lumen. TAP forms a heterodimer consisting of TAP1 and TAP2 subunits embedded in the ER membrane, each contributing six transmembrane domains and a nucleotide-binding domain for ATP hydrolysis.[25] Peptide binding occurs at a specific site in the ER-facing transmembrane domains, with TAP exhibiting selectivity for peptides bearing hydrophobic or basic residues at their C-terminus, ensuring compatibility with MHC class I groove preferences. ATP hydrolysis powers the conformational changes necessary for peptide transport across the membrane, with each cycle driven by the sequential binding and hydrolysis of two ATP molecules per subunit.[26]Upon binding, peptides are translocated into the ER at a rate of approximately 100 peptides per minute per TAP complex, enabling efficient supply for MHC class I loading. The affinity of peptides for TAP can be influenced by N-terminal trimming in the ER by endoplasmic reticulum aminopeptidase 1 (ERAP1), which processes longer precursors (often 9-16 residues) transported by TAP into optimal 8-10 residue lengths, thereby modulating the available peptide repertoire.[27] Viral pathogens have evolved mechanisms to evade this process; for instance, the human cytomegalovirus (HCMV) glycoprotein US6 binds to the ER-luminal side of TAP, inhibiting ATP binding and hydrolysis to block peptide translocation and reduce MHC class I surface expression.[28]
Peptide Loading and Editing
Peptide loading onto MHC class I molecules occurs in the endoplasmic reticulum (ER) following the translocation of cytosolic peptides via the transporter associated with antigen processing (TAP).[29] This process ensures that only high-affinity peptides, typically 8-10 amino acids long, are selected to stabilize the MHC class I complex for surface presentation. The peptide loading complex (PLC), a multi-protein assembly, orchestrates this selection by bridging TAP to newly synthesized MHC class I heavy chains associated with β2-microglobulin (β2m).[10]The PLC comprises tapasin, ERp57 (a protein disulfide isomerase), and calreticulin, which collectively chaperone MHC class I folding and peptidebinding. Tapasin, an ER-resident glycoprotein, recruits MHC class I to TAP, facilitating access to the peptide pool and promoting iterative peptideexchange to favor high-affinity ligands.[30] ERp57 and calreticulin assist in maintaining proper disulfide bonds and lectin-like binding to monoglucosylated MHC class I, respectively, enhancing the stability of the loading platform.[31] Concurrently, endoplasmic reticulum aminopeptidases (ERAP1 and ERAP2) trim the N-termini of imported peptides to optimize fit within the MHC class I binding groove, independently editing peptide length and sequence for better anchor residue compatibility.[32]During loading, empty MHC class I molecules are conformationally unstable and adopt an "open" form, exposing the peptide-binding groove for exchange, while high-affinity peptide binding induces a "closed" conformation that locks the complex.[33] Tapasin preferentially binds peptide-deficient MHC class I, catalyzing the removal of low-affinity peptides and replacement with superior ones through this conformational plasticity.[34] This editing ensures immunodominance of peptides with optimal binding kinetics.Quality control mechanisms retain unloaded or suboptimally loaded MHC class I in the ER via retention signals and chaperone interactions, preventing premature export.[35] Approximately 50% of MHC class I molecules are degraded in the ER if they remain peptide-free, primarily through ER-associated degradation (ERAD) pathways involving retrotranslocation to the cytosol and proteasomal proteolysis.[10] Only peptide-loaded complexes achieve sufficient stability for release from the PLC and progression to the Golgi.
Antigen Presentation Mechanism
Surface Expression and Stability
Following successful peptide loading within the peptide loading complex (PLC) in the endoplasmic reticulum (ER), MHC class I molecules dissociate from the PLC and are exported from the ER via COPII-coated vesicles.[36] This release typically occurs upon binding of high-affinity peptides, ensuring only stable complexes proceed in the secretory pathway.[36] The loaded MHC class I complexes then traverse the Golgi apparatus through the conventional secretory route, where the N-linked glycan on the heavy chain (at Asn86) undergoes maturation, including initial trimming of mannose residues by mannosidase I in the cis-Golgi compartment.[37] This processing step refines the high-mannose glycan acquired in the ER into complex forms as the molecules advance through the medial- and trans-Golgi networks.[37]From the trans-Golgi network, peptide-loaded MHC class I molecules are packaged into secretory vesicles and transported to the plasma membrane for insertion.[36] On the cell surface, these complexes achieve a typical density of approximately 10^5 to 10^6 molecules per cell, varying by cell type and physiological conditions.[2] This level of expression supports efficient antigen surveillance by cytotoxic T cells and natural killer cells.[38] Surface residency is not permanent; MHC class I molecules undergo constitutive endocytosis, primarily via clathrin-independent mechanisms involving Arf6, with rates influenced by their conformational stability.[39]The stability of surface MHC class I is predominantly governed by the affinity of the bound peptide, which dictates the complex's half-life, ranging from hours for low-affinity interactions to days for high-affinity ones.[39] Empty or peptide-receptive MHC class I molecules, lacking stable ligands, exhibit rapid internalization through endocytosis and are prone to degradation or retrieval, preventing unproductive surface presentation.[39] Following endocytosis, internalized complexes enter early endosomes for sorting: stable peptide-loaded forms are often directed to recycling endosomes (marked by Rab11a and Rab22a) for return to the plasma membrane, while unstable ones may proceed to late endosomes or lysosomes for degradation via the multivesicular body pathway.[39] In some cases, peptide-receptive MHC class I can be retrotranslocated from post-ER compartments back to the ER, mediated by chaperones like TAPBPR, allowing for peptide re-editing and potential reloading.[39]
Interaction with T Cell Receptor
The interaction between MHC class I molecules presenting antigenic peptides (pMHC) and the T cell receptor (TCR) on CD8+ T cells is a cornerstone of adaptive immune recognition. The structural basis of this interaction was first elucidated through X-ray crystallography of the human TCR A6 bound to HLA-A2 presenting the HTLV-1 Tax peptide, revealing a diagonal docking mode where the TCR sits atop the pMHC complex at an approximately 45-degree angle relative to the peptide-binding groove.[40] In this orientation, the complementarity-determining regions (CDRs) of the TCR α and β chains primarily contact the α1 and α2 helices of the MHC class I heavy chain, with CDR3 loops focusing on the exposed peptide residues to confer specificity. Additionally, the TCR α chain interacts with the peptide, while the β chain engages the MHC helices more extensively, enabling discrimination between self and foreign peptides.The binding interface extends beyond the TCR-pMHC contacts to include the CD8 co-receptor, which binds to the α3 domain of the MHC class I molecule, stabilizing the overall complex and facilitating signal transduction. The TCR α/β heterodimer makes direct contacts with the peptide-MHC via its variable domains, with the α3 domain serving as the primary docking site for the CD8 α/α or α/β homodimers or heterodimers expressed on cytotoxic T cells. This co-receptor engagement enhances the avidity of the interaction, as CD8 binding occurs independently of the TCR-pMHC contact but with distinct kinetics, exhibiting a low affinity (K_d ≈ 0.2 mM at 37°C) that supports rapid association and dissociation.[41] The combined TCR-pMHC and CD8-α3 interactions position the TCR for precise antigen surveillance on the cell surface.The affinity of TCR for pMHC complexes typically ranges from 1 to 100 μM, reflecting a balance between specificity and sensitivity that allows detection of rare antigens.[42] This moderate affinity arises from the structural complementarity at the interface, where germline-encoded polymorphisms in MHC class I alleles influence peptidepresentation and can lead to alloreactivity, as seen in transplant rejection where donor MHC variants are recognized as foreign by host T cells. Such alloreactivity stems from the mimicry of self-pMHC by allogeneic MHC loaded with self-peptides, driven by sequence differences in the α1 and α2 domains. The specificity is further tuned by the TCR's ability to cross-react with similar peptides, enabling broad immune coverage without excessive autoreactivity.[43]Upon pMHC engagement, TCR clustering on the T cell surface initiates signaling by recruiting and activating the Src family kinaseLck, which is associated with the CD8 co-receptor. This clustering amplifies weak individual interactions into a multivalent array, promoting Lck-mediated phosphorylation of immunoreceptor tyrosine-based activation motifs (ITAMs) on the CD3 ζ chains. The process aligns with the kinetic proofreading model, wherein multiple enzymatic steps impose a time delay, ensuring that only sustained TCR-pMHC engagements (lasting seconds to minutes) lead to productive signaling, while brief encounters dissociate without activation. This mechanism enhances discrimination between agonist and antagonist peptides, underpinning the specificity of T cell responses.[44]
Peptide Removal and Recycling
Peptide dissociation from MHC class I molecules occurs at a pH-dependent rate, with stable complexes exhibiting half-lives typically ranging from 1 to 10 hours at neutral pH on the cell surface.[45] In acidic endosomal environments (pH ≈5.0-6.0), dissociation accelerates significantly—up to 100-fold—due to stabilization of a peptide-empty intermediate, facilitating rapid off-rates without complete heavy chain-β2-microglobulin dissociation.[46][47] Surface stability, which correlates with peptideaffinity, influences removal rates, as more stable peptide-MHC complexes resist internalization and turnover.[45]Internalized peptide-MHC class I complexes are trafficked via clathrin-independent endocytosis to early sorting endosomes, where they may undergo sorting decisions.[48] A portion is directed to late endosomes and lysosomes for proteolytic degradation, while another fraction recycles back to the plasma membrane through Rab11-positive recycling endosomes, allowing reuse in antigen presentation.[48] This recycling pathway, involving GTPases like Arf6, enables dynamic turnover and contributes to the maintenance of surface peptide diversity.During recycling, dissociated peptides or newly generated fragments can undergo brief re-trimming by endosomal proteases, such as insulin-regulated aminopeptidase (IRAP), which removes N-terminal residues to optimize fit within the MHC class I groove. This editing process helps sustain a diverse and immunogenic peptide repertoire on recycled molecules, particularly in antigen-presenting cells.Unlike MHC class II, which relies on HLA-DM for catalyzed peptideexchange in endosomes, MHC class I lacks equivalent DM-like inhibitors or chaperones, making its endosomal editing primarily pH- and protease-driven.[2]
Immune Functions
Cytotoxic T Cell Activation
Cytotoxic T cells, also known as CD8+ T cells, are activated when their T cell receptor (TCR) recognizes antigenic peptides presented by MHC class I molecules on the surface of infected or abnormal cells. This TCR engagement provides signal 1 for T cell activation, but full activation requires co-stimulation through the CD28 receptor on the T cell interacting with B7-1 (CD80) or B7-2 (CD86) ligands on antigen-presenting cells, delivering signal 2. [49] Together, these signals trigger intracellular signaling cascades, including activation of NF-κB and NFAT pathways, leading to the production of interleukin-2 (IL-2) and expression of the IL-2 receptor alpha chain. IL-2 then drives clonal proliferation and differentiation of naive CD8+ T cells into effector cytotoxic T lymphocytes (CTLs). [50]Upon differentiation, CTLs acquire effector functions to eliminate target cells. The primary mechanism involves the release of cytotoxic granules containing perforin and granzymes through exocytosis at the immunological synapse. Perforin forms pores in the target cell membrane, allowing granzymes to enter and activate caspases, leading to apoptosis. Additionally, CTLs express Fas ligand (FasL), which binds Fas on target cells to induce death receptor-mediated apoptosis via the extrinsic pathway. These mechanisms ensure precise lysis of antigen-bearing cells while minimizing bystander damage.Effective CTL activation requires a threshold of TCR-pMHC interactions to initiate signaling above the activation threshold. This serial engagement model allows a single antigen-presenting cell to activate multiple T cells efficiently. Over the course of an immune response, CD8+ T cells undergo avidity maturation, where their functional sensitivity to peptide-MHC complexes increases up to 50-fold without necessarily selecting for higher-affinity TCRs, enhancing responsiveness to low-antigen levels. [50]Activated CD8+ T cells differentiate into memory subsets that provide long-term immunity. Central memory CD8+ T cells reside in lymphoid organs and exhibit high proliferative potential upon re-encountering antigen, while effector memory CD8+ T cells patrol peripheral tissues for rapid effector responses. These memory cells persist for years post-infection through homeostatic proliferation and IL-7/IL-15 signaling, enabling faster and more robust secondary responses. [51]
NK Cell Regulation
MHC class I molecules play a critical role in regulating natural killer (NK) cell activity through the "missing self" hypothesis, which posits that NK cells detect and eliminate cells lacking sufficient self MHC class I expression, thereby removing inhibitory signals that normally prevent NK-mediated cytotoxicity. This concept emerged from observations that NK cells reject tumor variants deficient in H-2 (the mouse equivalent of MHC class I) but spare those expressing normal levels, suggesting an alternative immune surveillance mechanism beyond adaptive responses. In healthy cells, surface MHC class I molecules engage inhibitory receptors on NK cells, setting a threshold for activation; their absence, as seen in virally infected or transformed cells, disarms this inhibition and licenses NK cell killing.[52]Central to this regulation are interactions between killer cell immunoglobulin-like receptors (KIRs) on NK cells and specific epitopes on HLA-C, the primary MHC class I ligand for human KIRs. HLA-C allotypes are grouped into C1 (characterized by asparagine at position 80) and C2 (lysine at position 80), with inhibitory KIR2DL2 and KIR2DL3 preferentially binding C1 epitopes, while KIR2DL1 binds C2 epitopes. Upon ligand engagement, these inhibitory KIRs transmit signals via immunoreceptor tyrosine-based inhibitory motifs (ITIMs) in their cytoplasmic tails, which recruit tyrosine phosphatases such as SHP-1, dephosphorylating activation signaling pathways and dampening NK cell responses like degranulation and cytokine production. This specificity ensures that NK cells from individuals expressing particular HLA-C groups are tuned to recognize deviations in self MHC expression.Balancing these inhibitory interactions, certain activating ligands on MHC class I molecules or non-classical variants can counteract inhibition under specific conditions. For instance, rare HLA alleles may engage activating KIRs like KIR2DS1, which shares structural similarity with KIR2DL1 but signals through ITAM-containing adaptors to promote NK activation. Non-classical MHC molecules, such as HLA-E, bind heterodimeric CD94/NKG2 receptors on NK cells; while the NKG2A isoform delivers inhibition similar to KIRs, the NKG2C isoform can activate NK cells, particularly in contexts like cytomegalovirus infection where HLA-E presents viral peptides. These activating interactions provide a rheostat-like balance, preventing over-inhibition while maintaining tolerance to healthy self cells.NK cell responsiveness is further calibrated during development through a process known as education or licensing, where interactions with self MHC class I ligands set the activation threshold for mature NK cells. NK cells expressing inhibitory receptors that bind self HLA molecules (e.g., KIR2DL1 in C2-positive individuals) become "educated" and hyporesponsive to targets lacking those ligands, ensuring functional competence only against truly abnormal cells. This tuning mechanism, observed in both human and mouse models, prevents autoimmunity by rendering uneducated NK cells anergic, while allowing educated ones to mount calibrated responses proportional to the strength of self MHC interactions during ontogeny.
Immune Surveillance Role
MHC class I molecules play a central role in immune surveillance by presenting endogenous peptides derived from intracellular proteins on the cell surface of nearly all nucleated cells, allowing cytotoxic CD8+ T cells to monitor for signs of cellular abnormality.[53] This presentation enables the immune system to discriminate between healthy self cells and those compromised by intracellular pathogens or oncogenic mutations, as altered protein degradation products—such as viral proteins or neoantigens from mutated genes—are loaded onto MHC class I for recognition.[54] In viral infections, for instance, peptides from pathogen-derived proteins are processed by the proteasome and displayed, signaling infection to trigger targeted cell lysis.[53] Similarly, in cancer, MHC class I showcases tumor-specific peptides arising from genetic alterations, facilitating immune detection and elimination of malignant cells.[54]Quantitative analyses of the MHC class I peptidome in infected cells reveal that viral peptides occupy a small but immunologically significant proportion of surface MHC class I molecules, sufficient to elicit robust T cell responses.[55] This low abundance underscores the sensitivity of the surveillance mechanism, where even minor shifts in peptide repertoire can alert patrolling CD8+ T cells to initiate protective cytotoxicity. In tumor contexts, analogous low-level presentation of neoantigens supports ongoing immune oversight, preventing unchecked proliferation.[54]A key extension of this surveillance is cross-presentation, primarily by dendritic cells, which internalize exogenous antigens—such as those from apoptotic infected cells or tumor debris—and process them for loading onto MHC class I via specialized pathways like endosome-to-cytosol translocation or vacuolar degradation.[56] This mechanism bridges extracellular threats to CD8+ T cell priming, enabling immune responses against viruses that evade direct infection of antigen-presenting cells or against non-replicating tumor antigens, thereby broadening systemic surveillance.[56]To prevent autoimmunity while maintaining vigilance, MHC class I contributes to central tolerance through AIRE (autoimmune regulator)-driven expression of tissue-specific self-antigens in medullary thymic epithelial cells, where these antigens are presented to developing thymocytes for negative selection of autoreactive CD8+ T cells.[57] This process ensures that only T cells tolerant to self-peptides mature, striking a balance that supports effective discrimination of non-self threats without aberrant self-attack.[57]
Specialized Physiological Roles
Maternal-Fetal Tolerance in Reproduction
In the context of maternal-fetal tolerance, extravillous trophoblast cells at the placental interface express the non-classical MHC class I molecule HLA-G, while avoiding expression of classical HLA-A, HLA-B, and HLA-C molecules, thereby minimizing recognition and attack by maternal cytotoxic T lymphocytes (CTLs).[58] This selective expression pattern is crucial for shielding the semi-allogeneic fetus from maternal alloreactive immune responses, as HLA-G's restricted presentation of peptides differs from the diverse repertoire of classical MHC class I, reducing the likelihood of triggering maternal T cell activation.[59] By limiting classical MHC class I on trophoblasts, the placenta establishes an immune-privileged environment that prevents graft-versus-host-like rejection of fetal tissues.[60]HLA-G exerts immunosuppressive effects by binding to inhibitory receptors such as immunoglobulin-like transcript 2 (ILT2) and ILT4 on immune cells, directly inhibiting the cytotoxic functions of both CTLs and natural killer (NK) cells at the maternal-fetal interface.[61] Specifically, HLA-G engagement with ILT2 on CD8+ T cells suppresses CTL proliferation and killing activity, while interaction with ILT2 and ILT4 on NK cells dampens their degranulation and cytokine release, collectively promoting tolerance without compromising broader antiviral defenses.[62] Recent research indicates that uterine natural killer (uNK) cells are educated by maternal MHC class I molecules, specifically through interaction of the NKG2A receptor with maternal HLA-E, promoting self-recognition and functional licensing that supports feto-placental development and lowers pre-eclampsia risk, as evidenced by large-scale genetic studies of over 150,000 pregnancies.[63] Additionally, the soluble isoform of HLA-G (sHLA-G), secreted by trophoblasts, further enhances tolerance by inducing the expansion and suppressive activity of regulatory T cells (Tregs), which modulate maternal immune responses to paternal antigens and support sustained immune homeostasis during gestation.[64]This expression profile represents an evolutionary adaptation in placental mammals, where the downregulation of classical MHC class I on trophoblasts reduces alloreactivity and fosters successful reproduction by evading maternal adaptive immunity.[65] Dysregulation of this system, such as aberrant HLA-G expression or altered interactions with maternal receptors, has been linked to increased risk of preeclampsia, a hypertensive disorder characterized by shallow trophoblast invasion and placental ischemia due to failed immune tolerance.[66] Supporting evidence from mouse models demonstrates that Qa-2, the murine homolog of HLA-G within the H2 complex, is essential for embryonic implantation and placental development; Qa-2-deficient mice exhibit intrauterine growth restriction and higher rates of fetal loss, underscoring its conserved role in maternal-fetal immune accommodation.[67]
PirB-Mediated Neural Plasticity
Major histocompatibility complex (MHC) class I molecules, traditionally known for their role in immune recognition, have been found to play an inhibitory function in neural plasticity through interaction with the paired immunoglobulin-like receptor B (PirB) in the mouse central nervous system. PirB, a receptor expressed on neurons, binds specifically to MHC class I ligands, thereby restricting synaptic remodeling in response to experience-dependent stimuli. This discovery was first reported in a study examining visual cortex development, where MHC class I expression on neurons was shown to signal via PirB to modulate plasticity during critical periods.The mechanism involves MHC class I proteins on neuronal surfaces engaging PirB, which activates downstream signaling pathways that suppress long-term potentiation (LTP), a key process in synaptic strengthening. Specifically, PirB engagement recruits phosphatases such as SHP-1 and SHP-2, which dephosphorylate and inhibit MAP kinase pathways essential for synaptic plasticity, thereby limiting the extent of LTP induction at hippocampal and cortical synapses. In the visual system, this interaction restricts ocular dominance plasticity, the ability of visual cortical neurons to shift preferences based on monocular deprivation during early development. Neurons lacking functional PirB exhibit enhanced LTP and prolonged critical periods for plasticity, allowing greater synaptic reorganization even in adulthood.[68][69]In PirB knockout mice, the absence of this inhibitory signaling leads to structural changes supporting heightened plasticity, including increased dendritic spine density and synapse formation in the visual cortex following sensory perturbations. These findings highlight PirB-MHC class I as a brake on neural adaptability, potentially to stabilize mature circuits after developmental windows close. For human relevance, PirB's ortholog, leukocyte immunoglobulin-like receptor B2 (LILRB2), shares structural and functional similarities, and modulating LILRB2 has been proposed to extend plasticity windows for treating amblyopia, a disorder of visual development where critical periods are limited. Blocking PirB in adult mice restores plasticity akin to juvenile levels, suggesting therapeutic potential for LILRB2-targeted interventions in humans to enhance recovery without traditional patching methods.[70]
Tissue-Specific Expression
MHC class I molecules, including the classical human leukocyte antigen (HLA) subtypes HLA-A, HLA-B, and HLA-C, are ubiquitously expressed on the surface of nearly all nucleated cells throughout the body, providing a foundational mechanism for antigen presentation to cytotoxic T cells. This broad distribution ensures continuous immune surveillance against intracellular pathogens and abnormal cells. In contrast, expression is absent on mature erythrocytes due to their anucleate nature, which precludes the synthesis of MHC class I proteins. Similarly, basal MHC class I levels on neurons are characteristically low, reflecting a specialized adaptation to minimize immune-mediated interference with neural function while maintaining responsiveness to inflammatory cues.[71][72][73]Expression of MHC class I is highly dynamic and inducible, particularly in response to inflammatory signals. Pro-inflammatory cytokines such as interferon-gamma (IFN-γ) and tumor necrosis factor-alpha (TNF-α) potently upregulate MHC class I on various cell types, including muscle fibers and endothelial cells, enhancing antigen presentation during infection or tissue damage. This induction occurs through transcriptional activation of MHC class I genes via cytokine-responsive elements, amplifying immune detection in inflamed tissues. Conversely, many tumors exhibit downregulated MHC class I expression as an immune evasion strategy, often mediated by epigenetic mechanisms involving histone deacetylases (HDACs) that repress gene transcription; HDAC inhibitors, such as vorinostat, can reverse this suppression by promoting chromatinacetylation and restoring surface expression on tumor cells.[74][75][76][77]Non-classical MHC class I molecules, including HLA-E, HLA-F, and HLA-G, display more restricted tissue-specific patterns compared to their classical counterparts. HLA-G is predominantly expressed on extravillous trophoblast cells at the maternal-fetal interface, contributing to immune tolerance during pregnancy, with minimal presence in other adult tissues. HLA-E shows limited distribution, primarily on activated immune cells and trophoblasts, while HLA-F expression is notably enriched in placental tissues, including cytotrophoblasts and syncytiotrophoblasts, where it supports trophoblast proliferation and invasion. These non-classical molecules often exhibit lower polymorphism and specialized ligand-binding properties tailored to their localized roles.[78]01756-7/fulltext)[79]During development, MHC class I expression undergoes significant changes to balance immune protection and tolerance. In early preimplantation embryos, classical MHC class I levels are low or undetectable, minimizing the risk of maternal immune rejection during the initial stages of gestation. Post-implantation, expression gradually increases in differentiating tissues, particularly in the trophoblast and fetal organs, coinciding with the establishment of the maternal-fetal interface and the onset of immune competence. This temporal regulation ensures that embryonic cells evade surveillance early on while acquiring the capacity for antigen presentation as development progresses.[72][80][81]
Pathogen Interactions
Viral Interference Mechanisms
Viruses have evolved diverse mechanisms to interfere with MHC class I antigen presentation, thereby evading recognition by cytotoxic T lymphocytes (CTLs) and promoting persistent infection. These strategies target various stages of the MHC class I pathway, including assembly, trafficking, and surface expression, often exploiting host cellular machinery for degradation or retention. Such evasions not only impair adaptive immunity but also alter interactions with innate effectors like natural killer (NK) cells.[82]One prominent approach involves direct inhibition of MHC class I surface expression. The HIV-1 accessory protein Nef downregulates MHC class I molecules by promoting their rapid endocytosis from the plasma membrane through a clathrin- and dynamin-independent pathway involving ADP-ribosylation factor 6 (ARF6), followed by sequestration in the trans-Golgi network or lysosomal degradation.[83] Similarly, human cytomegalovirus (HCMV) encodes US2 and US11 glycoproteins that cause ER retention and dislocation of newly synthesized MHC class I heavy chains to the cytosol, where they are targeted for proteasomal degradation via the ER-associated degradation (ERAD) pathway, requiring ubiquitination and interaction with Derlin-1.[84]Another strategy focuses on blocking peptide loading and transport in the ER. Adenovirus type 2 E19 protein binds directly to MHC class I heavy chains in the ER, retaining the complex via a dilysine ER retrieval motif and preventing association with the peptide transporter TAP, thus inhibiting maturation and surface transport.[85] Epstein-Barr virus (EBV) employs the BNLF2a protein, a short hydrophobic peptide, to inhibit TAP function by binding to its nucleotide-binding domain, thereby blocking ATP-dependent peptide translocation into the ER and reducing stable MHC class I-peptide complex formation on the cell surface.[86]Viruses also achieve global suppression of MHC class I expression by disrupting interferon (IFN) signaling pathways that normally upregulate antigen presentation. Measles virus (MV) V protein binds to STAT1 and STAT2, sequestering them in the cytoplasm and preventing their nuclear translocation upon IFN-α/β stimulation, which blocks the induction of IFN-stimulated genes including those enhancing MHC class I transcription and assembly. This suppression not only limits basal MHC class I levels but also prevents IFN-mediated amplification during infection.[87]These interference mechanisms carry significant consequences for host immunity, particularly increasing susceptibility to NK cell lysis due to diminished inhibitory ligands (e.g., HLA-A/B/C) on infected cells, activating the "missing self" recognition pathway.[82] However, viruses often counter this by additional evasions, such as HCMV UL18 mimicking MHC class I to engage inhibitory receptors. Over evolutionary timescales, such pressures have driven diversification; for instance, HIV clades exhibit sequence variations in Nef that modulate MHC class I downregulation efficiency, adapting to host HLA polymorphisms and contributing to clade-specific persistence.
Bacterial Modulation Strategies
Bacteria employ diverse strategies to modulate MHC class I antigen presentation, thereby evading cytotoxic T lymphocyte (CTL) recognition and promoting intracellular survival. Intracellular pathogens such as Listeria monocytogenes escape from the phagosomal vacuole into the host cytosol using the pore-forming toxin listeriolysin O, allowing replication in a compartment where proteins can be accessed by the proteasomal degradation pathway for MHC class I loading; however, this escape also enables L. monocytogenes to induce type I interferons (IFN-α/β), which sensitize infected macrophages to apoptosis and suppress IFN-γ-mediated activation, thereby reducing effective CTL responses.[88] Similarly, Mycobacterium tuberculosis resides within modified phagosomes that resist fusion with lysosomes and the endoplasmic reticulum (ER), limiting the delivery of phagosomal antigens to the cytosolic proteasomal machinery and ER peptide-loading complex, thereby impairing cross-presentation to CD8+ T cells in dendritic cells.[89]Surface-associated modulation occurs in extracellular bacteria like Neisseria gonorrhoeae, which adheres to epithelial cells and induces the shedding or internalization of host surface molecules, including HLA-A and HLA-B, through interactions with CEACAM receptors on antigen-presenting cells; this reduces MHC class I availability for CTL recognition and suppresses dendritic cell activation of antigen-specific CD8+ T cells.[90]The host counters these strategies through cytokine signaling, particularly IFN-γ, which induces the formation of immunoproteasomes by replacing constitutive proteasome subunits with immunosubunits (LMP2, LMP7, and MECL-1), enhancing the generation of peptides suitable for MHC class I binding and improving antigen presentation against intracellular bacteria like L. monocytogenes and M. tuberculosis.[24] This adaptive response restores efficient CTL activation, underscoring the dynamic interplay between bacterial evasion tactics and host immune countermeasures.
Therapeutic Implications for Infections
Therapeutic strategies targeting MHC class I have emerged as promising approaches to bolster CD8+ T cell responses against infectious diseases, particularly where pathogens evade immune surveillance by downregulating MHC class I expression. Peptide-based vaccines designed to mimic peptide-MHC class I complexes aim to directly stimulate cytotoxic T lymphocytes by delivering pathogen-derived epitopes that bind to patient-specific HLA alleles, thereby enhancing antigen-specific immunity without relying on endogenous processing. For instance, multi-epitope peptide vaccines targeting HIV-1 envelope glycoprotein gp120 have been developed to induce broad neutralizing antibodies and T cell responses, addressing the virus's high variability and immune escape mechanisms. These vaccines, often formulated with adjuvants to promote cross-presentation, have shown potential in preclinical models to restore MHC class I-restricted recognition in chronically infected cells. Similarly, peptide vaccines for hepatitis C virus (HCV) core and non-structural proteins have demonstrated induction of HLA-A2-restricted CD8+ T cells, offering a therapeutic avenue for persistent infections.Immune checkpoint inhibitors, such as anti-PD-1 antibodies, have been investigated to counteract T cell exhaustion in chronic viral infections, where prolonged antigen exposure leads to upregulated PD-1 expression on CD8+ T cells, impairing their recognition of MHC class I-presented viral peptides. In hepatitis B virus (HBV) infection, PD-1 blockade restores the functionality of exhausted HBV-specific CD8+ T cells, enhancing viral clearance in preclinical and early clinical trials; for example, nivolumab combined with therapeutic vaccines has achieved functional cures in some HBV-positive patients by reinvigorating MHC class I-restricted responses. As of 2025, combination therapies like VTP-300 therapeutic vaccine with nivolumab have shown sustained HBsAg reductions and functional cures in a subset of chronic HBV patients in phase II trials.[91] This approach leverages the natural role of MHC class I in presenting HBV epitopes to CD8+ T cells, countering the virus's partial evasion tactics like altered peptide processing. Clinical trials for chronic HBV and HIV have reported improved T cell proliferation and reduced viral loads with anti-PD-1 therapies, though risks such as HBV reactivation necessitate careful monitoring.Gene therapy strategies focus on restoring MHC class I expression in cells downregulated by viral interference, such as through adenoviral vectors delivering β2-microglobulin (β2M) to reconstitute functional MHC class I complexes on infected cell surfaces. In murine models of viral infection, β2M gene transfer has successfully upregulated MHC class I, enabling CD8+ T cell-mediated lysis of infected targets that would otherwise evade detection. Adaptations of chimeric antigen receptor (CAR) T cell therapy incorporating MHC class I-restricted T cell receptors (TCRs) target intracellular viral antigens presented by MHC class I, with preclinical studies demonstrating efficacy against HIV and cytomegalovirus by engineering patient-derived T cells to recognize specific pMHC complexes. These TCR-CAR constructs maintain HLA restriction, ensuring precise targeting while overcoming viral downregulation.Recent advances in the 2020s, informed by COVID-19mRNA vaccine platforms, have highlighted the potential of mRNA-based therapeutics to enhance MHC class I cross-presentation of viral antigens. SARS-CoV-2mRNA vaccines, such as those encoding spike protein epitopes optimized for HLA binding, promote efficient cytosolic translation and proteasomal processing in antigen-presenting cells, leading to robust CD8+ T cell priming via MHC class I. Insights from these vaccines have spurred designs incorporating MHC class I trafficking signals to boost cross-presentation of exogenous antigens, as seen in experimental mRNA constructs that elicit stronger T cell responses against influenza and other respiratory viruses. This approach not only accelerates vaccine development but also translates to therapeutic settings for chronic infections by amplifying MHC class I-dependent immunity.
Genetics and Polymorphism
Human HLA Genes and Loci
The human major histocompatibility complex (MHC) class I genes, known as human leukocyte antigen (HLA) genes, are located within the MHC locus on the short arm of chromosome 6 at position 6p21.3.[92] This locus encompasses a densely packed genomic region that includes the classical HLA class I genes HLA-A, HLA-B, and HLA-C, which span approximately 1.5 Mb and play central roles in antigen presentation to cytotoxic T cells.[93] These genes are oriented telomerically, with HLA-A positioned most distally, followed by HLA-H pseudogene, HLA-J pseudogene, HLA-G, HLA-F, HLA-E, and then HLA-C and HLA-B toward the centromere.[94]Each classical HLA class I gene consists of eight exons, encoding a heavy chain polypeptide that forms a heterodimer with β2-microglobulin.[4]Exon 1 encodes the leader peptide for signal sequence, exons 2 and 3 encode the α1 and α2 extracellular domains that form the peptide-binding groove, exon 4 encodes the α3 immunoglobulin-like domain for CD8 interaction, exon 5 encodes the transmembrane region, and exons 6, 7, and 8 encode cytoplasmic domains involved in intracellular signaling and stability.[4] This conserved exon-intron organization across HLA-A, HLA-B, and HLA-C facilitates the structural integrity of the MHC class I molecule, enabling peptide loading and surface expression.[95]Adjacent to the classical genes within the same chromosomal region are three non-classical HLA class I genes: HLA-E, HLA-F, and HLA-G, which exhibit limited polymorphism and specialized immunomodulatory functions.[71]HLA-E primarily presents signal peptides derived from classical MHC class I molecules to inhibit natural killer (NK) cell activity via CD94/NKG2 receptors, contributing to immune tolerance.[71] HLA-F, though less well-characterized, is implicated in interactions with NK cells and T cells during early pregnancy and viral infections, potentially modulating immune responses through inhibitory signaling.[71]HLA-G is predominantly expressed at the maternal-fetal interface, where it suppresses T cell and NK cell cytotoxicity to promote allograft tolerance, often via soluble isoforms that bind inhibitory receptors like LILRB1.[71]The immense diversity of HLA class I genes arises from extensive allelic variation, cataloged and standardized by the IPD-IMGT/HLA Database, the official repository for WHO-nominated sequences.[96] As of 2025, this database records over 29,000 alleles for HLA class I genes collectively, with HLA-A encompassing 8,949 alleles, HLA-B 10,680, HLA-C 8,944, HLA-E 385, HLA-F 126, and HLA-G 194; allelic nomenclature follows a systematic format (e.g., HLA-A*02:01:01) denoting gene, allele group, protein, and synonymous variants.[3] This polymorphism, concentrated in exons 2 and 3, underpins population-specific immune repertoires and transplant compatibility.[96]
Non-Human Isotypes and Alleles
In mice, the major histocompatibility complex (MHC) class I region, known as H2, encodes three classical loci: H2-K, H2-D, and H2-L, which present peptides to CD8+ T cells and exhibit haplotype-specific expression, with H2-L absent in certain strains like C57BL/6.[97] Non-classical isotypes in mice include those in the Qa (H2-Q) and Tla (H2-T) regions, comprising over 30 class Ib genes that display tissue-specific expression and limited polymorphism, often involved in specialized immune functions such as NK cell regulation.[98] These non-classical molecules, like Qa-1, are orthologous to humanHLA-E and bind nonamer peptides derived from classical MHC class I signal sequences to modulate immune responses.[99]Among non-human primates, the chimpanzee MHC class I genes are designated Patr-A, Patr-B, and Patr-C, serving as orthologs to human HLA-A, HLA-B, and HLA-C, respectively, with one functional copy per haplotype and an additional Patr-AL lineage in some individuals that resembles HLA-A.[100] In contrast, New World monkeys (Platyrrhini) exhibit expansions of MHC class I lineages, particularly G-like and B-like genes, arising from ancestral duplications that diversified after divergence from Old World monkeys, resulting in multiple paralogs such as Patr-G equivalents in species like the cotton-top tamarin.[101][102] These expansions contribute to species-specific allelic repertoires adapted to distinct pathogen pressures.Rodent MHC class I systems, exemplified by mice and rats, feature fewer polymorphic alleles at classical loci compared to primates—typically dozens rather than thousands—but compensate with higher gene copy numbers, including extensive class Ib duplications that enhance functional diversity through multigene families.[103] In birds, classical MHC class I genes are present but often reduced in number and organization compared to mammals; for instance, chickens possess only two dominantly expressed classical loci (BF1 and BF2) within a compact MHC, with passerines showing higher copy numbers and polymorphism in some lineages, though non-classical forms predominate in expression patterns.[104][105]In pigs, the swine leukocyte antigen (SLA) complex includes three classical MHC class I genes, SLA-1, SLA-2, and SLA-3, which are ubiquitously expressed and polymorphic, playing critical roles in immune recognition.[106] These loci are focal in xenotransplantation research, where human antibodies target SLA-1, -2, and -3, prompting genetic engineering strategies like CRISPR-mediated knockouts to generate MHC class I-null pigs that survive without eliciting acute rejection in preclinical models.[107][108]
Population Diversity and Typing
MHC class I molecules, encoded by human leukocyte antigen (HLA) genes, exhibit extensive polymorphism that varies across human populations, influencing immune responses to pathogens and disease susceptibility. For instance, HLA-B57 alleles, particularly HLA-B57:03, occur at higher frequencies in certain sub-Saharan African populations (approximately 5-10% in groups like South African Blacks) and confer protection against HIV-1 progression by restricting viral replication through cytotoxic T-cell responses.[109] In contrast, HLA-A*02, the most common HLA-A allele globally, is present at frequencies exceeding 20-50% in diverse populations, including Europeans, Asians, and Africans, contributing to broad peptide presentation capabilities.[110]These allelic distributions are tracked in resources like the Allele Frequency Net Database (AFND), which as of 2025 catalogs over 156,000 HLA allele frequencies from more than 14 million individuals across global populations, enabling comparative analyses of ethnic variations.[111] Such diversity often manifests in haplotypes with strong linkage disequilibrium (LD), where alleles at HLA-A, -B, and -C loci are inherited together more frequently than expected by chance, as seen in extended MHC blocks spanning hundreds of kilobases that preserve ancestral combinations in human populations.[112]Certain alleles are strongly associated with autoimmune diseases due to their role in antigen presentation. HLA-B*27, for example, is carried by 60-90% of patients with ankylosing spondylitis worldwide and increases disease risk up to 100-fold in carriers, likely through aberrant presentation of arthritogenic peptides that trigger inflammation.[113]HLA typing methods have evolved from serological assays, which detect surface antigens using antibodies but are now largely outdated due to limited resolution and inability to distinguish many alleles, to molecular techniques. Sanger sequencing provides allele-level resolution for exons 2-4 of HLA class I genes but is labor-intensive and costly for large-scale studies.[114] Next-generation sequencing (NGS) has become the gold standard for high-resolution typing, enabling full-length sequencing of HLA loci with accuracy >99% and throughput for population screening, as validated in transplant matching protocols.[115]
Evolutionary Aspects
Gene Duplication and Diversification
The multiplicity of MHC class I genes originated from ancient duplication events in the common ancestor of jawed vertebrates approximately 500 million years ago (Mya), when an ancestral peptide-binding gene underwent tandem duplications to form the proto-MHC cluster.[116] These early duplications established the tandemly arrayed structure of the MHC region, characterized by repeated blocks of class I genes interspersed with non-MHC genes, a pattern conserved across vertebrates from sharks to mammals.[117] This ancestral arrangement provided the genomic foundation for the adaptive immune system's antigen presentation capabilities, with subsequent expansions driven by segmental duplications that increased gene copy number and functional diversity.[117]In humans, the classical MHC class I genes HLA-A and HLA-B emerged through segmental duplications of non-classical precursors during early primateevolution, approximately 40–60Mya, while HLA-C arose later from the HLA-B lineage around 15 Mya.[118][119] These events involved large-scale genomic rearrangements in the MHC class I region on chromosome 6, where duplicated segments generated paralogous loci that diverged under selective pressures to specialize in presenting diverse peptides to cytotoxic T cells.[118] Non-classical genes like HLA-E and HLA-F, which arose from earlier duplications, retained more conserved functions, such as roles in NK cell regulation, whereas HLA-G emerged more recently; this highlights how duplication facilitated both innovation and specialization within the family.[117][119]Copy number variation (CNV) further contributes to MHC class I diversification, with some individuals carrying extra copies of pseudogenes such as HLA-H, a non-functional paralog derived from HLA-A.[120] This variability, often resulting from unequal crossing-over in the tandemly repeated MHC region, can expand or contract the total number of class I loci per haplotype, influencing the breadth of the presented peptide repertoire and immune responsiveness.[120] For instance, haplotypes with additional HLA-H copies may indirectly affect adjacent functional genes through linkage, altering antigen presentation diversity without producing viable proteins themselves.[121]Pathogen-driven selection has been a primary force in MHC class I gene diversification, promoting duplications and allelic variation to enhance host survival.[122]Heterozygote advantage arises because individuals with diverse MHC class I alleles can present a wider array of pathogen-derived peptides, evading immune escape by microbes and increasing resistance to infections.[123] This selective pressure is evident in higher MHC diversity in pathogen-rich environments, where duplication events amplify the genomic substrate for polymorphism, ensuring broader immune coverage across populations.[122]
Birth-and-Death Evolution Model
The birth-and-death evolution model posits that multigene families such as MHC class I evolve through a dynamic process of gene duplication, which generates new genes ("birth"), followed by divergence and potential loss via disabling mutations leading to pseudogenization or deletion ("death"). Proposed by Nei and Hughes in 1992, this model accounts for the observed polymorphism and turnover in MHC loci, where duplicate genes may either become fixed through positive selection or be eliminated if they confer no advantage or are deleterious.[124] Under this framework, the functional repertoire of MHC class I molecules is maintained despite high rates of gene gain and loss, allowing adaptation to diverse pathogens without concerted homogenization across family members.[125]Evidence supporting the model in MHC class I comes from phylogenetic analyses of primate lineages, where a substantial proportion of sequences represent pseudogenes, reflecting frequent gene death. For instance, in New World primates like the owl monkey, numerous MHC class I pseudogenes cluster with functional loci, indicating recent turnover events that align with the birth-and-death process.[102] Additionally, positive Darwinian selection drives rapid evolution at peptide-contact residues in the antigen-binding groove, as evidenced by dN/dS ratios exceeding 1 in these sites across human HLA class I loci, promoting diversification of peptide presentation capabilities.Balancing selection plays a key role in preserving polymorphisms under this model, with heterozygote advantage enabling individuals to recognize and respond to a wider array of pathogen-derived peptides compared to homozygotes. Pathogen-driven pressures further favor rare alleles through negative frequency-dependent selection, where less common variants evade prevalent pathogen evasion strategies, thereby sustaining allelic diversity over time.[126]In humans, the substitution rate at MHC class I loci approximates 10^{-9} per site per year, underscoring the relatively rapid allelic turnover consistent with birth-and-death dynamics.[127] This rate, combined with duplication events, facilitates ongoing evolution of the MHC class I family to counter shifting selective pressures from pathogens.[118]
Comparative Phylogeny Across Species
MHC class I molecules are conserved across all jawed vertebrates (gnathostomes), where they play a central role in antigen presentation to cytotoxic T cells as part of the adaptive immune system. This conservation extends from cartilaginous fish like sharks, which possess tightly linked class I genes with antigen-processing components such as TAP and LMP, to higher vertebrates. Recent studies have identified a primitive W-category of MHC molecules in cartilaginous fish, exhibiting features of both class I and II, suggesting an ancestral form from which modern class I diverged.[128] In contrast, jawless vertebrates (agnathans), including lampreys and hagfish, lack MHC class I and II genes entirely, relying instead on alternative adaptive immunity mechanisms like variable lymphocyte receptors (VLRs) for antigenrecognition.[129]In mammals, MHC class I gene repertoires exhibit significant expansions in certain lineages, particularly among artiodactyls. For instance, cattle (Bos taurus) harbor at least six classical class I loci (BoLA-1 through BoLA-6) alongside ten non-classical loci, totaling over ten class I genes per haplotype, which supports diverse peptide presentation tailored to pathogen pressures in ruminants. Conversely, monotremes such as the platypus (Ornithorhynchus anatinus) show contractions in their MHC class I repertoire, with classical class Ia genes colocalizing with class II and antigen-processing genes in a more compact arrangement reminiscent of non-mammalian vertebrates, reflecting an ancestral configuration with fewer duplicated loci.[130][131]Non-mammalian vertebrates display varied MHC class I architectures shaped by lineage-specific duplications. In amphibians like Xenopus (African clawed frog), the class I region is minimal, featuring a single classical class Ia locus closely linked to TAP and LMP genes, with additional non-classical class Ib genes located more distantly. Teleost fish, following their ancient whole-genome duplication approximately 350 million years ago, exhibit duplicated MHC class I genes, with classical loci often linked to antigen-processing machinery but unlinked from class II, enabling independent evolution and enhanced diversity in response to aquatic pathogens.[129][132]The ancient origins of MHC class I trace back to a duplication event from an ancestral class II-like gene around 450 million years ago, coinciding with the emergence of adaptive immunity in jawed vertebrates and subsequent adaptive radiation of immune gene families. This duplication established the foundational class I/II linkage observed in basal gnathostomes, with subsequent birth-and-death evolutionary dynamics driving species-specific diversification.[129]