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Lectin pathway

The lectin pathway is one of the three primary activation routes of the , a vital arm of the in vertebrates that enhances clearance and modulates . It is triggered when molecules, primarily carbohydrate-binding such as mannose-binding lectin (MBL) and ficolins, bind to specific sugar motifs on surfaces or damaged host cells, distinguishing it from the antibody-dependent classical pathway and the spontaneous alternative pathway. This recognition initiates a proteolytic cascade that converges with the other pathways at the formation of the , amplifying complement activation to promote opsonization, cell lysis, and immune cell recruitment. Key components of the lectin pathway include the recognition molecules—MBL, ficolins (ficolin-1, -2, and -3), and collectins like collectin-11 (CL-11) and collectin kidney 1 (CL-K1)—which form complexes with MBL-associated serine proteases (MASPs). Upon binding to pathogen-associated molecular patterns (PAMPs) or damage-associated molecular patterns (DAMPs), such as microbial glycans or acetylated groups, MASP-1 undergoes autoactivation and subsequently activates MASP-2, which cleaves complement proteins and to generate the C4bC2b (or C4b2a) . MASP-3 plays a supportive role, particularly in priming the alternative pathway, while regulatory MBL-associated proteins (MAps), such as MAp19 and MAp44, help fine-tune the response. The pathway's activation is tightly regulated to prevent host tissue damage, primarily through inhibitors like the C1 inhibitor (C1-INH), antithrombin, and factor I, which target MASP activity and C4b degradation. Dysregulation of the lectin pathway has been implicated in various diseases, including infections, autoimmune disorders like IgA nephropathy, ischemia-reperfusion injury, and rheumatic heart disease, highlighting its dual role in protection and pathology. Beyond immunity, emerging evidence suggests non-canonical functions in embryonic development and tumor suppression, underscoring its broader physiological impact.

Overview

Introduction

The lectin pathway is a key activation route within the , functioning as an innate immune mechanism that initiates complement cascade through the recognition of specific patterns on surfaces or damaged cells. This pathway enables rapid host defense by binding pattern recognition molecules to microbial structures, thereby triggering proteolytic events that amplify immune responses without the need for antibodies or prior exposure to antigens. The discovery of the lectin pathway occurred in the 1990s, building on earlier identification of mannose-binding protein (now termed mannose-binding or MBL) in during the 1980s, with pivotal studies demonstrating its role in complement activation analogous to the classical pathway's C1q. Seminal research, including work by Ikeda et al. in 1987 on MBL's association with complement activity and subsequent findings by Matsushita and Fujita in on associated serine proteases, established the pathway's distinct initiation mechanism. Further confirmation in 1997 by Thiel et al. highlighted the involvement of these proteases in cleaving key complement components, solidifying the lectin pathway as a dedicated arm of innate immunity. In host defense, the lectin pathway bridges the gap in innate immunity by directly sensing evolutionarily conserved microbial motifs, such as residues, thereby facilitating early clearance independent of adaptive responses. This recognition-driven activation underscores its evolutionary importance in providing immediate protection against infections. The pathway culminates in the deposition of fragments for opsonization, generation of anaphylatoxins like C5a to induce and immune cell recruitment, and ultimately the formation of the membrane attack complex for .

Comparison with other pathways

The lectin pathway differs fundamentally from the other two complement activation pathways in its initiation, being antibody-independent and relying on soluble such as mannose-binding lectin (MBL) or ficolins to recognize pathogen-associated molecular patterns (PAMPs) like carbohydrates on microbial surfaces. In contrast, the classical pathway requires antibodies (IgG or IgM) bound to antigens, forming immune complexes that engage C1q for activation. The alternative pathway initiates through spontaneous hydrolysis of in , leading to low-level deposition on surfaces and amplification via factor B and D, without needing prior immune recognition. Despite these differences, all three pathways share downstream elements, converging at the formation of a that cleaves into C3a and C3b, thereby initiating opsonization, anaphylatoxin release, and progression to the terminal membrane attack complex () for cell . The lectin and classical pathways both generate the C4b2a convertase, while the alternative uses C3bBb, but the subsequent cascade from onward is identical across pathways. Evolutionarily, the lectin pathway is considered an ancient innate immune mechanism, with homologs of its recognition molecules and serine proteases present in and protochordates, predating the adaptive immunity-linked classical pathway and the more recently evolved pathway in vertebrates. This underscores its role as a primordial defense against pathogens, maintained through vertebrates for rapid, non-specific responses.
PathwayInitiatorsTriggersKey Regulators
ClassicalC1q, C1r, C1sImmune complexes, apoptotic cellsC1-INH, C4BP
LectinMBL, ficolins, MASPsCarbohydrates, PAMPs on pathogensC1-INH, MAp44/MAp19, C4BP
C3, factor B, factor DSpontaneous C3 , surfacesFactor H, factor I, , MCP

Key Components

Mannose-binding lectin (MBL)

Mannose-binding lectin (MBL), also known as mannose-binding protein, is a key pattern recognition molecule in the that initiates the lectin pathway of complement activation. It circulates in as a soluble protein capable of recognizing specific structures on the surface of pathogens, thereby facilitating immune defense without prior . MBL is structurally and functionally part of the collectin family, which includes other proteins like proteins A and D, sharing features that enable multivalent binding to microbial surfaces. The structure of MBL consists of multiple identical polypeptide chains, each approximately 32 kDa, that assemble into a bouquet-like configuration. Each chain features an N-terminal cysteine-rich domain for oligomerization, followed by a collagen-like stalk that forms rigid triple helices, a neck region, and a C-terminal carbohydrate-recognition domain (CRD) responsible for ligand binding. The basic structural unit is a trimer of three chains forming a triple helix via the collagenous region, stabilized by interchain disulfide bonds and hydrophobic interactions; these trimers further oligomerize into higher-order multimers, typically ranging from dimers to hexamers of trimers (equivalent to 6-18 chains), with the higher multimers exhibiting greater functional avidity for pathogen recognition. MBL is primarily synthesized in the liver by hepatocytes and secreted into the bloodstream, where it maintains concentrations of 0.5-5 μg/mL in healthy individuals. As a member of the collectin family, it is evolutionarily conserved across vertebrates and plays a non-redundant role in innate immunity by binding to terminal , , or residues in oligomannose or hybrid-type glycans commonly found on the surfaces of , fungi, yeasts, and certain viruses, but rarely on host cells due to differences in patterns. This binding promotes pathogen opsonization for , agglutination to limit spread, and recruitment of effector molecules; upon ligand engagement, MBL associates with MBL-associated serine proteases (MASPs) to trigger downstream complement activation. Genetically, MBL is encoded by the MBL2 gene located on the long arm of chromosome 10 (10q11.2-q21). The gene contains four s, with 1 encoding the CRD and collagen-like regions, and polymorphisms in this —particularly the single nucleotide variants rs1800450 ( B, Gly54Asp), rs1800451 ( C, Arg52Cys), and rs5030737 ( D, Gly57Glu)—disrupt the collagen domain's assembly, leading to reduced oligomerization and low MBL levels (often <0.1 μg/mL in homozygous or compound heterozygous individuals). These structural variants, collectively termed the O s (in contrast to the wild-type A ), occur at frequencies of 5-15% in various populations and are associated with MBL deficiency, which affects up to 10% of individuals and may influence susceptibility to infections, though clinical impacts vary by context. Promoter region polymorphisms (e.g., rs7095891 and rs7096206) further modulate expression levels.

Ficolins

Ficolins serve as alternative molecules in the lectin pathway of the , distinct from mannose-binding lectin by their reliance on fibrinogen-like domains for recognition rather than domains. Humans express three types of ficolins: L-ficolin (also known as ficolin-2), M-ficolin (ficolin-1), and H-ficolin (ficolin-3). These proteins initiate upon to pathogen-associated molecular patterns, contributing to innate immunity. L-ficolin and H-ficolin are primarily synthesized in the liver and secreted into the , while M-ficolin is constitutively expressed on the surface of immune cells such as monocytes, macrophages, and neutrophils, with lower levels detectable in . L-ficolin is also found in the liver, and H-ficolin additionally appears in tissues, bile ducts, and alveoli. This differential expression allows ficolins to function both systemically and at local sites of or . Structurally, ficolins feature an N-terminal cysteine-rich region, a central collagen-like domain composed of glycine-X-Y repeats that facilitates oligomerization, and a C-terminal fibrinogen-like (FBG) domain responsible for ligand binding. The collagen-like regions enable the formation of higher-order oligomers, typically assembling into bouquet-like structures of 4–6 trimers, which enhance for multivalent targets on microbial surfaces. Unlike collectins, the FBG domain in ficolins replaces the carbohydrate-recognition domain, allowing recognition of non-carbohydrate motifs. Ficolins exhibit broad specificity for acetylated groups, such as (GlcNAc) and (GalNAc), found on bacterial cell walls, fungal surfaces, and apoptotic cells. For instance, L-ficolin binds GlcNAc, GalNAc, and N-acetylmannosamine, recognizing pathogens like and , as well as DNA on damaged cells. M-ficolin targets GlcNAc and , interacting with microbes such as and . H-ficolin recognizes GlcNAc, D-fucose, and , aiding in the clearance of late apoptotic cells and pathogens including and Salmonella typhimurium. These binding events lead to recruitment of MBL-associated serine proteases (MASPs), mirroring the activation mechanism of mannose-binding lectin.

Collectin liver 1 and kidney 1

Collectin liver 1 (CL-L1, also known as collectin-10) and collectin kidney 1 (CL-K1, also known as collectin-11 or CL-11) are additional molecules in the lectin pathway, belonging to the collectin family like MBL. They often form heteromeric complexes, primarily in a 1:2 ratio of CL-L1 to CL-K1 subunits, which circulate in serum at concentrations of approximately 0.3–1.5 μg/mL for the complex. Structurally, both CL-L1 and CL-K1 share the collectin architecture: an N-terminal cysteine-rich domain, a collagen-like region for oligomerization, a neck domain, and a C-terminal domain for carbohydrate binding. The heterocomplex assembles into bouquet-like multimers similar to MBL, enabling high-avidity binding to microbial surfaces. CL-L1 is predominantly expressed in the liver, while CL-K1 is highly expressed in the kidneys, but both are found in various tissues including endothelial and epithelial cells, with presence indicating systemic roles. These collectins recognize a variety of ligands, including high-mannose oligosaccharides, GlcNAc, and fucose on bacteria, fungi, and viruses, as well as DNA and stress-induced patterns on damaged host cells. Upon binding, the CL-L1/CL-K1 complex associates with MASPs, particularly MASP-1 and MASP-3, to activate the lectin pathway, contributing to complement-mediated clearance in infections and tissue injury. Mutations in the COLEC10 (CL-L1) and COLEC11 (CL-K1) genes are linked to the rare developmental disorder 3MC syndrome, underscoring their roles beyond immunity in embryonic development.

MBL-associated serine proteases (MASPs)

MBL-associated serine proteases (MASPs) are a family of enzymes that associate with mannose-binding lectin (MBL) or ficolins to initiate the lectin pathway of complement activation. These proteases bridge the recognition of pathogen-associated molecular patterns by to the proteolytic cascade of the . The family includes three active serine proteases—MASP-1, MASP-2, and MASP-3—and two inactive forms known as MAp19 (also called MASP-2 fragment or ) and MAp44 (also known as MAP-1). The MASPs exhibit a modular structure characteristic of the C1r/C1s/sea urchin epidermal growth factor/bone morphogenetic protein-1 (C1r/C1s-UMB) family of serine proteases. Each active MASP consists of a heavy chain (comprising CUB1, EGF-like, CUB2, CCP1, and CCP2 domains) disulfide-linked to a light chain containing the serine protease (SP) domain, which is responsible for catalytic activity. MASP-1 and MASP-3 are produced by alternative splicing of the MASP1 gene, sharing identical heavy chains but differing in their SP domains, while MASP-2 and the truncated MAp19 arise from the MASP2 gene; MAp19 lacks the SP domain and consists only of the CUB1-EGF-like regions followed by a short unique sequence. MAp44 is an alternative splice product of the MASP1 gene, comprising the heavy chain domains without the SP domain, and circulates at higher concentrations (around 200–800 ng/mL) than other MASPs. These domains enable calcium-dependent binding to MBL or ficolins, forming heterotetrameric or higher-order complexes analogous to the C1 complex in the classical pathway. Activation of MASPs occurs through autoactivation triggered by conformational changes upon lectin binding to carbohydrate surfaces. MASP-1 undergoes rapid autoactivation and subsequently activates MASP-2, which then primarily cleaves and to propagate the pathway. MASP-3, despite its structural similarity, does not appear to autoactivate in the same manner and its precise role remains under investigation, potentially involving alternative complement pathways. MAp19, being enzymatically inactive, may serve a regulatory function by competing for binding sites on lectins without contributing to . Similarly, MAp44 acts as an endogenous by binding to MBL and ficolins, thereby competing with active MASPs and dampening lectin pathway activation to prevent excessive . Evolutionarily, the MASPs are homologous to the C1r and C1s proteases of the classical complement pathway, sharing not only the domain architecture but also a common ancestral origin that underscores the structural and functional parallels between the lectin and classical pathways. This homology is evident in the conserved CUB-EGF-CUB modules that facilitate dimerization and lectin association, as well as the SP domains that enable substrate recognition and cleavage.

Activation Process

Pattern Recognition

The lectin pathway of the is initiated through by soluble , primarily mannose-binding lectin (MBL) and ficolins, which serve as molecules that detect pathogen-associated molecular patterns (PAMPs) on microbial surfaces. These bind to specific carbohydrate structures, such as terminal residues in mannans on cell walls or N-acetylglucosamine in lipopolysaccharides (LPS) on , thereby distinguishing microbial threats from host components. This binding event triggers the assembly of higher-order complexes essential for downstream signaling. The specificity of MBL and ficolins arises from their ability to recognize non-self carbohydrates that are typically exposed on pathogens but masked on host cells by terminal residues. MBL preferentially targets arrays of , , or in a calcium-dependent manner, while ficolins, such as L-ficolin and M-ficolin, recognize acetylated groups like or GlcNAc-containing motifs prevalent on bacterial surfaces. This selective binding avoids activation by the densely sialylated glycans of mammalian cells, ensuring targeted immune responses against foreign invaders. Upon binding, multivalent PAMPs on surfaces induce clustering and oligomerization of MBL and ficolins, forming stable multimers that create a scaffold for the of MBL-associated serine proteases (MASPs) as downstream effectors. This structural rearrangement enhances and facilitates the initiation of the proteolytic cascade. For instance, MBL oligomers can accommodate multiple MASP dimers, amplifying the signal for complement activation. Representative examples of this recognition include MBL binding to high-mannose glycans on the envelope glycoprotein gp120, which facilitates viral opsonization and complement-mediated neutralization, and ficolin-2 interacting with O-acetylated capsular on Streptococcus pneumoniae serotype 11A, promoting bacterial clearance via the lectin pathway. These interactions highlight the pathway's role in defending against diverse pathogens, from viruses to encapsulated bacteria.

Proteolytic Cascade

Upon binding of pattern recognition molecules such as mannose-binding lectin (MBL) or ficolins to -associated molecular patterns, the associated serine proteases are activated, initiating the proteolytic of the pathway. MASP-1 undergoes autoactivation first due to its higher catalytic efficiency (k_cat/K_m = 4.5 × 10² M⁻¹ s⁻¹), followed by its activation of MASP-2 with a rate of k_cat/K_m = 1.2 × 10⁴ M⁻¹ s⁻¹, as MASP-2 exhibits low autoactivation (k_cat/K_m = 0.14 M⁻¹ s⁻¹). This sequential activation ensures efficient propagation on localized pathogen surfaces. Activated MASP-2 then cleaves the complement component into the anaphylatoxin C4a and the larger fragment C4b, with high catalytic efficiency (k_cat/K_m = 2.97 × 10⁷ M⁻¹ s⁻¹). The C4b fragment covalently binds to nearby surfaces, including carbohydrates or proteins, via its reactive group, anchoring the cascade to the activation site and preventing . This surface-bound C4b serves as a platform for subsequent steps. Next, MASP-2 proteolytically activates by cleaving it into the small fragment C2b and the larger subunit C2a. The C2a fragment associates with surface-bound C4b to form the (C4b2a). MASP-1 supports this process not only by activating MASP-2 but also by directly cleaving , providing an auxiliary pathway for C2 activation. Additionally, MASP-1 exhibits D-like activity, contributing to cross-talk with the alternative pathway through limited of complement components. The proteolytic cascade amplifies rapidly on pathogen surfaces due to the high local concentrations of activators and substrates, enabled by intercomplex activation among MASP oligomers and the surface-restricted deposition of fragments like C4b. This kinetic advantage, driven by enzymatic efficiencies orders of magnitude higher than solution-phase reactions, ensures swift complement deposition and opsonization.

Formation of C3 Convertase

The formation of the C3 convertase in the lectin pathway occurs following the activation of mannose-binding lectin (MBL)-associated serine protease-2 (MASP-2), which cleaves C4 into C4a and C4b, with C4b covalently binding to the pathogen surface via its thioester bond, and cleaves C2 into C2a and C2b, allowing the C2a fragment to associate with surface-bound C4b to assemble the C4b2a complex. In this complex, C4b serves as the substrate-binding subunit that recruits and positions C3 for cleavage, while C2a provides the catalytic serine protease site responsible for the hydrolytic activity. The C4b2a functions by cleaving the central complement component into C3a, an anaphylatoxin that promotes by inducing mast cell degranulation and chemotaxis, and C3b, a key that facilitates by marking the target surface for immune cell recognition. The generated C3b molecules deposit covalently onto nearby surfaces through their reactive , amplifying localized complement activation while minimizing systemic effects. For stability and efficiency, the C4b2a complex is primarily surface-bound, which enhances its activity on pathogen membranes and restricts diffusion, but it decays rapidly in the fluid phase due to the instability of the C4b-C2a interaction, preventing uncontrolled complement consumption. Unlike the alternative pathway's (C3bBb), which forms spontaneously on host or pathogen surfaces without prior specific and relies on factor B for , the lectin pathway's C4b2a is initiated by of carbohydrates and incorporates C4b for targeted assembly, ensuring antibody-independent but lectin-dependent activation.

Amplification and Effector Functions

Upon activation of the in the lectin pathway, the resulting C3b fragments deposit on surfaces, initiating an amplification loop through crossover to the pathway. The deposited C3b binds factor B, forming a pro-convertase complex (C3bB) that is cleaved by factor D into Bb, generating the C3 convertase C3bBb. This complex cleaves additional C3 molecules, producing more C3b and exponentially enhancing complement deposition on the target surface for efficient immune clearance. The amplification further progresses with the association of surface-bound C3b to the lectin pathway (C4b2a), forming the C4b2a3b. This enzyme specifically cleaves into the anaphylatoxin C5a, which acts as a potent chemoattractant for neutrophils and promotes , and C5b, which initiates the terminal pathway. C5b sequentially binds and C7 to form the C5b67 complex, which inserts into the target membrane; C8 then joins to facilitate the of multiple C9 molecules, assembling the membrane attack complex (, or C5b-9). The pore-forming MAC disrupts the membrane integrity, leading to cell and destruction. Beyond lysis, the lectin pathway effectors mediate opsonization and inflammatory responses. C3b covalently attaches to microbial surfaces, serving as an that binds complement receptor 1 (CR1) on such as macrophages and neutrophils, facilitating enhanced and immune complex clearance. Additionally, the anaphylatoxins C3a and C5a bind their respective G-protein-coupled receptors (C3aR and C5aR) on immune cells, triggering , release, and to amplify local and recruit effector cells to the infection site.

Regulation

Regulatory Proteins

The lectin pathway of the complement system is tightly controlled by a suite of regulatory proteins that prevent excessive activation and potential host tissue damage. These include both soluble and membrane-bound factors that target key enzymes and activation products, ensuring pathway initiation and amplification occur only at appropriate sites and intensities. Among the primary regulators are C1 inhibitor (C1-INH), C4-binding protein (C4BP), membrane cofactor protein (MCP), and factor H, each exerting specific inhibitory effects on the pathway's proteolytic components. C1-INH serves as the principal soluble inhibitor of the lectin pathway by directly binding to and inactivating the mannose-binding lectin-associated serine proteases (MASPs), particularly MASP-1 and MASP-2. This interaction forms stable complexes that halt the autoactivation of MASPs and block their proteolytic activity, thereby preventing the initial cleavage of complement component into C4a and C4b. As a family member, C1-INH's stoichiometric inhibition is crucial for maintaining , with its plasma concentration typically exceeding that of MASPs to ensure rapid quenching of activation signals. Deficiency or dysfunction in C1-INH can lead to dysregulated lectin pathway activity, underscoring its central role. C4BP functions as a key fluid-phase regulator that modulates the lectin pathway at the level of the (C4b2a). Composed of multiple alpha and beta chains forming a large oligomeric structure, C4BP binds avidly to nascent C4b fragments generated by MASP-2, serving as a cofactor for factor I-mediated cleavage of C4b into inactive forms. This binding not only facilitates C4b degradation but also accelerates the intrinsic decay of the C4b2a complex by promoting the dissociation of the C2a subunit, thereby limiting convertase stability and downstream amplification more stringently in the lectin pathway compared to the classical pathway. C4BP's association with further enhances its regulatory efficiency on activated surfaces. MCP, also known as , is a widely expressed transmembrane that acts as a surface-bound cofactor to regulate the lectin pathway on host cells. It binds to C3b and C4b deposited during activation, recruiting factor I to proteolytically inactivate these opsonins and prevent convertase reassembly. Through its four short consensus repeats, MCP specifically targets the C3b component of the (C3bBb or C4b2a in cross-talk scenarios), promoting its cleavage at multiple sites to terminate amplification. This protective mechanism is essential for distinguishing self from non-self surfaces, with MCP's expression on most nucleated cells providing localized control. Another membrane regulator is (DAF, also known as CD55), which binds to C4b and C3b to accelerate the dissociation of the C4b2a and C3bBb convertases, further protecting host cells from complement activation. Factor H, the predominant soluble regulator of complement, plays a supportive but limited role in the lectin pathway primarily through fluid-phase modulation of C3 activation products. As a 155-kDa , it acts as a cofactor for factor I in degrading fluid-phase C3b generated via lectin pathway amplification, thereby dampening systemic spread of activation. also competitively inhibits formation by binding C3b and displacing essential substrates, though its affinity is higher for alternative pathway surfaces; in the lectin context, this contributes to overall without directly targeting MASPs. Its multifaceted interactions with glycosaminoglycans and sialic acids further aid in restricting lectin-mediated responses to sites. MBL-associated proteins (MAps), such as MAp19 and MAp44, serve as non-catalytic inhibitors specific to the lectin pathway. MAp44, an alternative splice product of the MASP1/3 gene, competes with MASP-2 for binding to molecules like MBL and ficolins, thereby inhibiting complex formation and autoactivation to set an activation threshold. MAp19, derived from the MASP2 gene, similarly competes with MASP-2 for binding but has a lower affinity, and its inhibitory role remains somewhat debated. Antithrombin provides an additional soluble inhibitory mechanism by binding and inactivating MASP-1 and MASP-2, particularly in the presence of , linking complement regulation to control.

Inhibitory Mechanisms

The lectin pathway of the is tightly regulated to prevent excessive on host tissues, primarily through mechanisms that target the of convertases, proteolytic of activation fragments, and controlled initiation of the . One key inhibitory process involves decay acceleration, where C4b-binding protein (C4BP) binds to C4b within the C4b2a, promoting the dissociation of the catalytically active C2a subunit and thereby accelerating the decay of the complex. This action significantly reduces the of the C4b2a convertase, limiting downstream amplification in both the classical and lectin pathways. Additionally, (C1-INH) plays a role by forming complexes with MASP-2, thereby inhibiting its proteolytic activity early in the . Proteolytic inactivation represents another critical regulatory step, mediated by factor I, a that cleaves the alpha chains of C4b and C3b to generate inactive fragments, preventing their incorporation into convertases or opsonization. This cleavage requires cofactors such as membrane cofactor protein (MCP) or complement receptor 1 (CR1), which bind to C4b or C3b on host cell surfaces, positioning them for factor I-mediated degradation and thereby protecting self-cells from complement-mediated damage. These cofactors enhance the specificity and efficiency of inactivation in the lectin pathway, ensuring that activation fragments deposited on host surfaces are rapidly neutralized. Threshold control further safeguards against unwarranted activation, as MASP-1 and MASP-2 exhibit low autoactivation rates in the absence of binding to pattern recognition molecules like MBL or ficolins, thereby avoiding spontaneous initiation of the cascade on host surfaces. This intrinsic low activity of MASPs maintains the pathway in a quiescent state until microbial carbohydrates are detected, minimizing the risk of . Host protection is bolstered by the concentration of regulatory proteins on self-cells, facilitated by interactions with residues that mask mannose-like patterns and prevent recognition by lectin pathway initiators such as MBL. on cell surfaces sterically hinder binding of MBL and ficolins, directing regulators like C4BP and factor I to accumulate where needed, thus preferentially inhibiting complement activation on healthy host tissues while allowing response to foreign .

Clinical Significance

Deficiencies and Diseases

Deficiencies in components of the lectin pathway can impair innate immune responses, leading to increased susceptibility to infections and, in some cases, autoimmune conditions. deficiency, arising from polymorphisms in the , affects up to 30% of individuals in certain populations, resulting in low serum MBL levels. This genetic variation is associated with recurrent infections, particularly in children, including respiratory and invasive bacterial infections during the first decade of life when adaptive immunity is still maturing. Additionally, MBL deficiency has been linked to an elevated risk of autoimmune diseases such as , where low MBL levels correlate with increased disease severity and susceptibility to infections in affected patients. Ficolin deficiencies are rarer than MBL variants, with homozygous mutations in ficolin genes occurring infrequently and often resulting in undetectable serum levels of specific ficolins like ficolin-3. Variants in L-ficolin (ficolin-2), a key pattern recognition molecule in the lectin pathway, have been associated with increased risk of infections, including infections and in vulnerable populations such as neonates. Furthermore, low circulating levels of L-ficolin or ficolin-3 during are linked to , potentially due to dysregulated complement activation contributing to and . MASP-2 deficiency, caused by mutations in the MASP2 gene, impairs the proteolytic activation of and in the lectin pathway, preventing efficient formation of the . Individuals with this deficiency exhibit recurrent severe infections, such as pneumonias and other bacterial s, highlighting its role as a in some cases. Overactivation of the lectin pathway can exacerbate tissue damage in inflammatory conditions. In ischemia-reperfusion injury, excessive lectin pathway activation following restoration of blood flow promotes endothelial damage and inflammation, contributing to in settings like or transplantation. Similarly, dysregulated lectin pathway activity in amplifies systemic inflammation, leading to endothelial barrier disruption and worsened outcomes in severe s. These pathological effects parallel broader complement deficiencies, which broadly increase infection risk across pathways.

Therapeutic Implications

The lectin pathway of the has emerged as a promising for therapeutic in conditions involving dysregulated complement , particularly where overactivation contributes to . C1-esterase (C1-INH) concentrates, such as Cinryze, are approved for the prophylaxis and treatment of (HAE), a condition linked to C1-INH deficiency that can lead to lectin pathway overactivation due to uninhibited MASP-1 and MASP-2 activity. Cinryze replenishes functional C1-INH levels, thereby suppressing lectin pathway initiation and reducing angioedema attacks, with clinical trials demonstrating significant reductions in attack frequency and severity. Targeting MASP-2, the key protease in the lectin pathway's formation, offers more selective inhibition. Narsoplimab, a against MASP-2, has shown efficacy in a pivotal phase 2 trial for transplant-associated (HSCT-TMA), a life-threatening complication driven by endothelial damage and complement dysregulation, with a 61% overall response rate in adults. Excellent outcomes have also been reported in pediatric patients from programs. The biologics license application () for narsoplimab in HSCT-TMA was resubmitted to the FDA in May 2025 following a Complete Response Letter and is currently under review, with a PDUFA target action date of December 26, 2025, highlighting its potential as a disease-specific therapy. Prospects for in lectin pathway disorders focus on correcting MBL2 polymorphisms that cause mannose-binding (MBL) deficiency and associated immunodeficiencies. Preclinical studies in murine models have demonstrated that nonviral gene transfer of MBL DNA via tail-vein injection can achieve sustained high MBL levels, restoring lectin pathway function and enhancing opsonization against pathogens. Such approaches hold promise for addressing recurrent infections in MBL-deficient individuals, though clinical translation remains in early stages with no active trials as of 2025. Serum MBL levels serve as valuable biomarkers for risk stratification in lectin pathway-related conditions, guiding therapeutic decisions in infections and . Low serum MBL concentrations, often due to MBL2 variants, correlate with increased susceptibility to severe infections, such as and pneumococcal disease, enabling identification of at-risk patients for prophylactic interventions. In autoimmune contexts, elevated or dysregulated MBL levels have been linked to disease progression in conditions like systemic lupus erythematosus, supporting their use in monitoring complement involvement and tailoring therapies.

References

  1. [1]
    The Lectin Pathway of the Complement System—Activation ... - MDPI
    Among the three main activation routes of complement, the lectin pathway (LP) has been discovered the latest, and it is still the subject of intense research.
  2. [2]
    Complement activation, regulation, and molecular basis for ...
    In the lectin pathway, patterns of glycans are detected via MBL, CL‐LK, or ficolins leading to activation of MASPs and formation of the same C3 convertase, ...<|control11|><|separator|>
  3. [3]
  4. [4]
  5. [5]
  6. [6]
  7. [7]
  8. [8]
  9. [9]
    Mannose‐binding lectin in innate immunity: past, present and future
    The existence of mammalian serum lectins was first predicted in 1975 by Robinson et al. (5), and the protein was first isolated in 1978 from cytosolic fractions ...
  10. [10]
    Mannose-binding lectin: biology and clinical implications - PubMed
    The basic structural unit is a triple helix of MBL peptides, which aggregate into complement-fixing higher-order structures (tetramers, pentamers and hexamers) ...
  11. [11]
    Mannose-Binding Lectin: Biologic Characteristics and Role in ... - NIH
    Three polypeptide chains form a triple helix through the collagenous region, stabilized by hydrophobic interaction and interchain disulphide bonds within the N- ...
  12. [12]
    Mannose-binding lectin and the balance between immune ...
    Structurally, MBL comprises a cysteine-rich domain at the N-terminus followed by a collagen-like domain, a neck region and a carbohydrate recognition domain ( ...
  13. [13]
    Mannose‐Binding Lectin: Biologic Characteristics and Role in the ...
    Jan 26, 2017 · MBL binding causes these microorganisms to agglutinate and allows phagocytic clearance of pathogens as well as lectin-complement pathway ...2. Mbl: Protein And Biologic... · 3.1. Mbl2 Gene Polymorphisms · 5. Mbl: Clinical...
  14. [14]
    The mannose-binding lectin: a prototypic pattern recognition molecule
    The mannose-binding lectin (MBL) is an evolutionary conserved circulating host defense protein that acts as a broad-spectrum recognition molecule against a wide ...
  15. [15]
    Mannose-binding lectin gene polymorphisms are associated with ...
    May 15, 2002 · MBL2 polymorphisms resulting in low serum levels are present in at least 30% of individuals.8-10 MBL deficiency appears to be an important ...
  16. [16]
    Impact of Exon 1 polymorphism in the MBL2 gene on MBL serum ...
    Jan 2, 2025 · Polymorphisms in the MBL2 gene exon 1 can decrease serum levels of mannose-binding lectin (MBL), increasing the risk of infection in immunocompromised ...
  17. [17]
    Mannose-binding lectin: structure, function, genetics and disease ...
    Mannose-binding lectin (MBL), a serum protein characterised by both collagenous regions and lectin domains, plays an important role in innate immune defence.
  18. [18]
    Ficolins and the Recognition of Pathogenic Microorganisms - NIH
    Feb 5, 2019 · Ficolins are innate pattern recognition receptors (PRRs) similar to the collectin, the mannose-binding lectin (MBL), and the surfactant proteins ...
  19. [19]
    Structural and functional diversity of collectins and ficolins and their ...
    Sep 11, 2017 · Ficolins contain both a collagen-like domain and a fibrinogen-like domain that has a specific binding affinity for N-acetylglucosamine. They can ...
  20. [20]
    Structural insights into the innate immune recognition specificities of L
    Ficolins exhibit a similar oligomeric structure, but their collagen-like stalks are followed by a domain homologous to the fibrinogen β and γ chains (Matsushita ...L-Ficolin Recognizes... · L-Ficolin Binds To A Wide... · Ficolins As Novel Types Of...
  21. [21]
    MBL-associated serine proteases (MASPs) and infectious diseases
    In this review, we summarize the findings on the role of MASP polymorphisms and serum levels in bacterial, viral and protozoan infectious diseases.
  22. [22]
    Humoral Pattern Recognition and the Complement System
    May 14, 2013 · Activation of the lectin pathway occurs through direct recognition of carbohydrate or acetylated PAMPs by MBL and ficolins, respectively, in ...<|control11|><|separator|>
  23. [23]
    Ficolins: Novel pattern recognition molecules of the innate immune ...
    Ficolins are collectin proteins that recognize pathogen-associated patterns, activating the immune system and activating the complement cascade.
  24. [24]
    New Insights into the Role of Ficolins in the Lectin Pathway of Innate ...
    Ficolin-2 was identified as a new type lectin, which recognizes carbohydrates such as N-acetylglucosamine (GlcNAc) in sugar chains. The lectin activity of ...
  25. [25]
    Glycans as a key factor in self and nonself discrimination: impact on ...
    Apr 5, 2022 · Glycans have an enormous power to discriminate self/nonself as they constitute a fundamental molecular ID of both human cells and pathogens.
  26. [26]
    Be on Target: Strategies of Targeting Alternative and Lectin Pathway ...
    It is probable that low oligomeric MBL and ficolins bind a single MASP dimer, while higher oligomers (pentamers, hexamers) can bind two MASP dimers ...Complement-Mediated Diseases · Masp-1 Is Required For Ap... · Active Masp-3 Is The...
  27. [27]
    Molecular defects in the mannose binding lectin pathway in ...
    Mar 25, 2010 · Upon binding to PAMPs, the MBL pathway and MASP recruitment are activated. MASP-2 is analogous to C1s as it forms C3 convertase by sequentially ...
  28. [28]
    Mannan-binding lectin activates C3 and the alternative complement ...
    May 1, 2006 · Lectin pathway activation of C3 is known to involve target recognition by mannan-binding lectin (MBL) or ficolins and generation of classical pathway C3 ...
  29. [29]
    Mannose Binding Lectin (MBL) and HIV - PubMed - NIH
    A number of studies have clearly shown that MBL binds to HIV. Binding of MBL to HIV is dependent on the high-mannose glycans on gp120.
  30. [30]
    Ficolin-2 Lectin Complement Pathway Mediates Capsule-Specific ...
    Mar 27, 2022 · We demonstrate the human lectin ficolin-2 binds to Streptococcus pneumoniae serotype 11A capsule polysaccharide dependent on the O-acetyltransferase gene wcjE.
  31. [31]
    The Lectin Pathway of the Complement System—Activation ...
    Among the three main activation routes of complement, the lectin pathway (LP) has been discovered the latest, and it is still the subject of intense research.
  32. [32]
    The complement system and innate immunity - Immunobiology - NCBI
    The mannan-binding lectin pathway (MB-lectin pathway) is initiated by binding of the mannan-binding lectin, a serum protein, to mannose-containing carbohydrates ...2-5. Complement is a system... · 2-9. Hydrolysis of C3 causes...
  33. [33]
    Essential role of Mannose-binding lectin-associated serine protease ...
    It is clear that MASP-1 is an essential protease of both the lectin and alternative complement pathways.
  34. [34]
    Complement and its role in innate and adaptive immune responses
    Dec 15, 2009 · The complement system plays a crucial role in the innate defense against common pathogens. Activation of complement leads to robust and ...
  35. [35]
    Lectin Pathway - an overview | ScienceDirect Topics
    The first lectin recognition molecule discovered was mannose-binding ... This pathway is initiated by binding of a lectin, the mannose binding protein ...
  36. [36]
    Complement component C3 - The “Swiss Army Knife” of innate ...
    Importantly, formation of C3b enables the binding of the protease Factor B (FB), and the resulting pro-convertase (i.e. C3bB) is quickly transformed by Factor D ...
  37. [37]
    The complement system: history, pathways, cascade and inhibitors
    The complement system is an integral part of the innate immune response and acts as a bridge between innate and acquired immunity.
  38. [38]
    How is Complement Activated in Lectin Pathway - Creative Biolabs
    Recognition of Pathogen Surfaces. The lectin pathway is activated when MBL or ficolins bind to specific carbohydrate structures on the surface of pathogens.Missing: specificity | Show results with:specificity
  39. [39]
    Complement Membrane Attack Complex: New Roles, Mechanisms ...
    All three pathways converge on the C3b-mediated proteolytic cleavage of C5 to form C5a and C5b and the C5b-mediated sequential assembly of C5b-7, C5b-8, and MAC ...
  40. [40]
    Purified complement C3b triggers phagocytosis and activation of ...
    Jan 6, 2023 · We show that purified C3b can potently trigger phagocytosis and killing of bacterial cells via Complement receptor 1.
  41. [41]
    Article Structure of the C3b Binding Site of CR1 (CD35), the Immune ...
    It fulfills this role through binding C3b/C4b-opsonized foreign antigens. The ... Binding of CR1 to C3b/C4b is also critical to the complement system.
  42. [42]
    Clinical manifestations of mannan-binding lectin deficiency - PMC
    In SLE patients, MBL deficiency increase the risk for respiratory tract infections (Garred et al., 2001, Takahashi et al., 2005) as well as the risk of ...
  43. [43]
    Low Levels of Mannan-Binding Lectin or Ficolins Are Not Associated ...
    Thus, whereas ficolin deficiencies are rare, MBL deficiencies may constitute ... (2005) Human M-ficolin is a secretory protein that activates the lectin ...
  44. [44]
    Two factors of the lectin pathway of complement, l-ficolin and ...
    We found relative l-ficolin deficiency to be associated with prematurity, low birthweight and infections. l-Ficolin concentration correlated with ...Missing: preeclampsia | Show results with:preeclampsia
  45. [45]
    Circulating ficolin-2 and ficolin-3 in normal pregnancy and pre ... - NIH
    According to our findings, pregnant women with low circulating levels of ficolin-2 or ficolin-3 have an increased risk for pre-eclampsia. Low ficolin-2 and ...
  46. [46]
    Disease-Causing Mutations in Genes of the Complement System
    MASP-1 and MASP-2 are responsible for complement activation through cleavage of C2 and C4 (Figure 2B). The cascade then proceeds as described above for the ...
  47. [47]
    Mannan-binding lectin-associated serine protease-2 (MASP-2 ... - NIH
    Mar 24, 2014 · Two cases of MASP-2 deficiency previously reported by us were a 12-year-old boy with recurrent pneumonias and a 4-year-old girl with recurrence ...
  48. [48]
    Mannan binding lectin-associated serine protease-2 (MASP-2 ...
    Aug 30, 2016 · Complement activation via the lectin activation pathway (LP) has been identified as the key mechanism behind post-ischemic tissue inflammation ...Missing: sepsis | Show results with:sepsis
  49. [49]
    The Role of Complement System in Septic Shock - PMC - NIH
    Activation of these two pathways is essential to clear endotoxin. Recent investigations have shed light on the role of lectin complement pathway in septic shock ...<|control11|><|separator|>
  50. [50]
    Infections of People with Complement Deficiencies and Patients ...
    Activation of complement in the fluid phase can occur through the classical, lectin, or alternative pathway. Deficiencies of components of the classical pathway ...<|control11|><|separator|>
  51. [51]
    Cinryze, a Human Plasma-Derived C1 Esterase Inhibitor for ... - NIH
    Cinryze is the first FDA-approved C1 esterase inhibitor therapy for preventing HAE attacks in the US.
  52. [52]
    Complement Blockade With a C1 Esterase Inhibitor In Paroxysmal ...
    Nov 15, 2013 · C1 INH inhibits the classical pathway of the complement system by binding C1r and C1s and inhibits the mannose-binding lectin-associated serine ...
  53. [53]
    Safety and efficacy of narsoplimab in pediatric and adult patients ...
    May 21, 2024 · Transplant-associated thrombotic microangiopathy (TA-TMA) is a severe complication following hematopoietic stem cell transplantation (HSCT).
  54. [54]
    Narsoplimab Results in Excellent Survival in Adults and Children ...
    Aug 29, 2025 · Narsoplimab Results in Excellent Survival in Adults and Children With Hematopoietic Cell Transplant Associated Thrombotic Microangiopathy (TA- ...
  55. [55]
    Omeros Announces Publication Highlighting Survival Outcomes in ...
    Sep 2, 2025 · A biologics license application (BLA) for narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic ...
  56. [56]
    Levels of mannose-binding lectin (MBL) associates with sepsis ...
    Aug 12, 2020 · Low serum mannose-binding lectin level increases the risk of death due to pneumococcal infection. Clin Infect Dis. 2008;47:510–6. PubMed ...Missing: autoimmunity | Show results with:autoimmunity
  57. [57]
    MBL Deficiency as Risk of Infection and Autoimmunity - PMC
    MBL deficiency is genetically determined and predisposes to recurrent infections and chronic inflammatory diseases.