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Chapter 23: Immune Response & Lymphocyte Biology

Immune System – Core Principles & Architecture

  • The immune system’s primary duty: discriminate “self” vs. “non-self” to prevent infection and malignancy while avoiding auto-aggression.

    • Immunologic tolerance - absence of destructive antiself immune responses

    • Autoimmunity -failure of self vs non self discrimination, or failure of immunologic tolerance

  • Failure of discrimination ➜ autoimmunity; failure of tolerance induction especially critical at the B- and T-cell developmental checkpoints.

  • Innate vs. adaptive immunity distinctions (Table 23-1)

    • Innate: germ-line encoded receptors, polymorphonuclear phagocytes, NK, eosinophils; rapid non-clonal response

      • NK cells - principal component of innate immunity

    • Adaptive: rearranged antigen-specific receptors on B & T lymphocytes; clonal, delayed but specific, generates memory; mediated by the recognition of cell surface proteins encoded by major histocompatibility complex (MHC) in the short arm of chrom 6

      • MHC class I and MHC class II or Human leukocyte antigens (HLA class I and HLA class II)

        • Encodes several secreted proteins of the complement system - TNF

    • Non-self discrimination - adaptive immune response characterized by immunologic specificity and memory

      • Acquired resistance of to one antigen does not confer resistance to another unrelated (non—cross-reactive) antigen

      • Anamnestic or secondary immune response - cells previously exposed to and specific for one foreign protein or antigen respond more rapidly and more effectively to subsequent exposure to that same antigen

        • Humoral immunity - antibodies produced by B lymphocytes and their progeny

        • Cell-mediated immunity - mediated by T lymphocytes

Lymphocyte Ontogeny

  • All lymphocytes derive from CD34⁺ hematopoietic stem cells (HSCs) in fetal liver ➜ post-natal bone marrow.

    • CD34⁺ - gives rise to both myeloid and lymphoid early progenitors; involved in cell adhesion and important for inhibition of hematopoietic differentiation

  • Commitment sequences (murine & human data)

    • HSC ➜ Common Lymphoid Progenitor (CLP) ➜ B, T, NK; Myeloid CMP ➜ GMP / MEP.

    • Lineage commitment evidence: single-cell HSC division yields one repopulating HSC + one myeloid-restricted daughter (murine).

  • Resting B and T cells circulate in quiescent state and function only after encountering cognate antigen

    • Both T and B cells require costimulatory signals in the form of cell surface receptor engagement or soluble factors, for effector function

    • Antigen presenting cells (APCs) - accessory cells of innate immune system which mediates recognition of antigen by T cells and the costimulatory signals for both B and T cells

  • Each lymphocyte has only 1 specific antigen to which it can respond

    • T cell - unique T cell receptor (TCR); recognizes small processed oligopeptides bound within the cleft of an MHC on the surface of an APC

      • APC - Dendritic cells, B cells, monocytes-macrophages, epithelial Langerhans cells and endothelial cells

    • B cells - unique Ig displayed on the surface as B cell receptor (BCR) or be secreted

B-Cell Development (Fig 23-2 & 23-4)
  • B cell maturation occurs in the bone marrow -HSC committed to B lymphoid lineage develop into immature IgM+ and IgD- B cells (1st phase of B cell maturation)

    • Immature B cells released from the bone marrow complete their development in the spleen and differentiate into:

      • Follicular B cells

      • Marginal B cells

  • Most critical step - expression of Ig molecules on the surface as BCR, specificity is same as the antibody that will be secreted by the activated mature B cell progeny

  • Bone-marrow phases

    • Pro-B: CD34⁺CD19⁺CD10⁺

    • Pre-B: expression of \mu heavy chain + surrogate light chain (VpreB + λ5) = pre-BCR; Bruton’s tyrosine kinase (Btk) crucial; boys with Btk mutation ➜ X-linked agammaglobulinaemia - normal pro-B cells but lack pre-B cells and their progeny

    • Immature B: express surface IgM (sIgM) with Igα/Igβ (CD79a/b), down-regulate CD34/CD10, up-regulate CD20/CD22

      • sIgM+, CD19+, CD10+, and CD20+ but have not acquired IgD expression which occurs after peripheral maturation

  • Light-chain rearrangement: variable region of the κ gene then λ loci if κ non-productive.

  • Central tolerance: deletion/receptor-editing of high-affinity self-reactive clones (55-75 % of human immature B cells self-reactive).

  • Peripheral maturation in spleen

    • Naive mature B cells - resting at G0 phase, express CD19 and CD20 and sIgM

      • Maturation involves:

        • positive selection - recognize antigen

        • negative selection - eliminate B cells whose BCR recognize self-antigens with high affinity; deletion of potentially autoreactive clones and induction of B cell tolerance

    • immature B cells - CD10-, IgD+

    • Follicular B vs. Marginal-zone B determination influenced by BCR signal strength.

Immunoglobulin Structure (Fig 23-3)
  • Two 25 kDa light (κ/λ) + two 50-70 kDa heavy chains.

  • Variable (V), Diversity (D, heavy only), Joining (J) gene segments create V(D)J diversity; CDR3 hypervariable most diverse.

  • Five heavy‐chain classes: IgM, IgG (1-4), IgA (1-2), IgD, IgE – each with Fc-mediated effector functions.

    • Defined by expression of the heavy chain: \mu,\gamma,\alpha,\delta,\varepsilon

    • Ig heavy chains contain sites for binding of Fc receptors (FcR) and complement

    • IgM - 1st class of antibody to be produced during development

    • IgD - marker for later B cell deveopment

    • IgA - secreted as dimer, found at mucosal surfaces such as the intestine

    • IgG - bulk of circulating Ig

  • Genes encoding the heavy chains of Ig are located in chrom 14 whereas genes encoding light chains on chrom 2 and 22

  • Earliest B cell precursor express pan B cell marker CD19 and early marker CD10

T-Cell Development (Fig 23-5)
  • Differentiation occurs largely in the thymus; derived from self renewing pluripotent HSCs

    • Bulk of peripheral T cells - either CD4+ helper or CD8+ cytolytic lineages

    • CD34+ cells subdivided by expression of CD38 and MHC-like molecule CD1a

    • Earliest thymocytes reside in the cortex - CD34+, express pan T cell markers such as CD2 and CD7 but lack TCR-associated αβ heterodimer and CD3-associated subunits of the TCR complex and the helper and cytotoxic mature T cell coreceptors CD4 and CD8 ➜ CD3-,CD4-,CD8-, or triple negative

  • Thymic stages: CD3⁻CD4⁻CD8⁻ triple-negative (TN) ➜ β-selection (pre-TCR) ➜ CD4⁺CD8⁺ double positive (DP) ➜ Positive & Negative selection ➜ lineage commitment to CD4 or CD8 single-positive (SP).

    • CD34+, CD38-, CD1a- ➜ CD34+, CD38+, CD1a- ➜ CD34+, CD38+, CD1a+

    • Acquisition of CD1a - associated with loss of NK, dendritic cell and plasmacytoid dendritic cell differentiation

    • CD1a+ cells - express recombinase-activating gene products RAG1 and RAG2 and early TCR rearrangements detectable after acquisition of CD38 and CD1a

  • TCR - receptor for antigen recognition

  • Hallmark of cellular immune response - antigen specificity - conferred by rearranged and selected TCR on the surface of T lymphocytes

  • TCR gene clusters: α, β, γ, δ – serial rearrangement; TRECs from δRec-ΨJα excision serve as thymic output markers (neonatal SCID screening).

  • Positive selection: rescue DP that recognize self-MHC + self-peptide with low/intermediate affinity.

  • Negative selection: deletion of high-affinity self-reactive clones; AIRE gene in medullary TECs promotes ectopic tissue-antigen expression – mutation ➜ APECED.

Major Histocompatibility Complex (MHC)
  • MHC class I - major determinants are HLA-A, HLA-B and HLA-C

    • Ensure variation in the affinity of peptide binding; any single microbe is unlikely to mutate such that it is unable to bind all MHC molecules in the population and therefore escape T cells recognition

  • MHC class II - major determinants are HLA-DR, HLA-DQ and HLA-DP

    • Determines the affinity and specificity of peptide binding and T cell recognition; expressed only in B cells, monocyte-macrophages, and dendritic cells

    • May be induced on the surface of monocyte-macrophages, fibroblasts, endothelia cells, and certain mesenchymal and epidermal cells by variety of inflammatory mediators and cytokines such as IFN-γ.

    • Can be inducibly expressed only T lymphocytes thus rendering T cells capable of antigen presentation to other T cells

  • Class I (HLA-A/B/C): expressed on all nucleated cells, present 8-11 aa peptides to CD8; α chain + β2-microglobulin.

  • Class II (HLA-DR/DQ/DP): on APCs, present 12-20 aa peptides to CD4; αβ heterodimer.

  • Drug hypersensitivity (abacavir/HLA-B57:01; carbamazepine/HLA-B15:02) due to altered peptide repertoire (“p-i concept”).

Peripheral Lymphocyte Activation

B-Cell Activation
  • TI-1 antigens (e.g., LPS via TLRs) ➜ polyclonal IgM.

  • TI-2 antigens (repetitive polysaccharides) ➜ marginal-zone B; class switching aided by cytokines.

  • TD antigens: need cognate T cell help (CD40–CD40L, cytokines IL-4/IL-21) ➜ Germinal Center reaction: somatic hypermutation (AID-dependent), affinity maturation, class switch recombination, memory (CD27⁺) & plasma cell differentiation (CD138⁺).

  • BCR signaling: Igα/Igβ ITAMs phosphorylated by Lyn/Fyn/Blk ➜ Syk, BLNK, Btk, PLCγ2 ➜ DAG/IP₃ ➜ PKCβ, Ca²⁺ rise, MAPK, NF-κB, NF-AT.

T-Cell Activation
Cytotoxic Mechanisms
  • CD8⁺ CTL & NK: perforin/granzyme and Fas–FasL.

  • Defects (PRF1, UNC13D, STX11, STXBP2) ➜ Familial hemophagocytic lymphohistiocytosis.

Spleen – Anatomy & Immunologic Function

  • Critical secondary lymphoid organ with both immunologic and nonimmunologic function

  • Identified in the human embryogenesis during week 5 of gestation; weighs about 10g, then 30g at 1 year of life, 60g by 5yo

  • Receives 3% to 5% of the total cardiac output

  • White pulp - collections of lymphocytes and macrophages that form small white nodules

    • Periarteriolar lymphoid sheath (PALS) - circumferential ring of leukocytes that surround the central arterioles; primarily T lymphocytes;

    • Spheroidal lymphoid follicles - arise at intervals from PALS; primarily B lymphocytes; IgM-IgD+CD1c-CD23+

      • Primary follicle - naïve mature B cells that coexpress IgM and IgD; CD27-

      • secondary follicles - contain activated B cells within its germinal center (GC)

    • marginal zone - positioned around the follicles and not in direct contact with red pulp - IgM⁺IgD⁻ CD1c⁺ CD23- B cells and high levels of CD21

    • Marginal zone-like B cells - circulate blood with memory phenotype IgM+IgD+CD27+

  • Red pulp cords (Billroth) filter & groom erythrocytes; removal of inclusions (Howell-Jolly bodies) & senescent cells.

    • Closed (rapid) splenic circulation - erythrocytes pass directly from arterial to venous circulation by way of traditional endothelialized capillaries — primarily nutritive

    • Open (slow) circulation - allows careful filtration of individual erythrocytes, with repair and phagocytosis of cells that cannot successfully negotiate the return trip

Important functions:

  • initiates immune response for T and B lymphocyte response and proliferation

    • Polysaccharide antigens - require this unique immunologic environment for optimal antibody response

    • Intravenouse antigens - found during infection or after immunization

    • Early IgM response and secondary IgG response

  • Bloodborne bacteria, encapsulated organisms, are removed by splenic macrophages located in the PALS and in the cords

  • Nonimmunologic functions:

    • Efficient filter for erythrocytes and is able to perform grooming (removal of antibodies or other surface molecules), culling (destruction of cells), and pitting functions (removal of intracellular material)

    • Erythrocytes with intracellular inclusions - nuclear remnants or micronuclei (Howell-Jolly bodies), denatured hgb (Heinz bodies), siderotic granules (Pappenheimer bodies), malarial parasites

    • Hypersplenism - enlarged spleen trapping a larger portion of circulating blood cells

  • Abnormalities of the spleen:

    • Congenital Asplenia and Polysplenia

      • Ivemark Syndrome -body has bilateral right sidedness with trilobed lungs and a centralized liver; cardiac defects are common and risk of infection

        • Deletion of HOX11 ; heterozygous inactivating mutations in RPSA (ribosomal protein SA)

      • Accessory spleen and ectopic spleens

    • Splenosis and Splenoptosis

      • Wandering spleen - not fixed within the retroperitoneum; can be palpated anywhere in the abdomen

      • Splenosis - autotransplantation of splenic tissue into the ommentum or peritoneal surfaces of the abdominal cavity; occurs after fracture or rupture of the spleen

    • Splenic Sequestration

      • Enlargement of the spleen that occurs when blood enters the organ but is unable to exit properLy

      • Observed in sickle cell anemia when venous return of blood is hindered by the intrasplenic sickling or erythrocytes within the red pulp

    • Hypersplenism

      • Splenomegaly with non-immune-mediated trapping of peripheral blood cells and results in mild to moderate cytopenia

  • Splenectomy indications: trauma, hereditary spherocytosis, ITP/AIHA; subtotal splenectomy preserves immune function.

  • Overwhelming Postsplenectomy infection (OPSI) risk with encapsulated bacteria ➜ lifelong vaccination & prophylactic antibiotics.

B-Cell Activation and Function

  • trigger for clonal expansion of the responding B cell

  • Memory cells - rapidly respond on reencounter with the same antigen

  • Plasma cells - terminally differentiated B cells found primarily in the bone marrow specialized for high level secretion of IgG, IgM, and IgA; continuously secrete antibodies

  • Affinity maturation - isotype switching from low affinity IgM to high affinity IgG, IgA, or IgE and somatic hypermutation

  • Recognition of antigen by BCR occurs with or without T cell help

    • Thymus dependent (TD) - modulated and enhanced cell-cell contact with T cells

    • Thymus independent (TI)

Thymus-Dependent vs Thymus Independent

BCR - capable of binding a variety of antigens, including soluble proteins and polysaccharides; recognition requires cooperation with T cells, or T cell help or T cell dependent (TD);

  • Naive follicular B cells in lymph node follicles or the spleen recognize their cognate antigen while contacting a helper T cell specific for the same antigen

  • Germinal center (GC) - where isotype switching, somatic hypermutation, memory cell generation, and plasma cell differentiation occur

  • T1 antigens

    • T1-1 antigens - bacterial components that can trigger B cell activation regardless of Ig specificity and lead to polyclonal activation

      • Pathogen-associated molecular pattern (PAMP) - activate B cells by binding to a distinct family of receptors, TLRs accounting for their independence from Ig; ie: lipopolysaccharide (LPS)

      • Ig secreted in response to T1-1 is low affinity IgM because T1-1 antigen binding does not induce class switching

    • T1-2 antigens - molecules that have repetitive structure and activate B cells by cross-linking of multiple BCRs on a single cell leading to specific clonal antibodies production; bacterially derived polysaccharides such as in encapsulated organisms

      • marginal zone B cells are important for responses to T1-2 antigens

      • Major site - spleen; antigen produced is often IgM

      • Class switching does not depend on Tcell help but is enhanced by noncontact T cell factors such as IL-4

T-Cell Activation and Function

CD8+ cytolytic T cells - induce the lysis of foreign cells, such as infected, malignant, or allogeneic cells

CD4+ helper T cells - interact with B cells via cell-cell and soluble factors to induce class switching and the generation of antigen-specific antibodies; provide “help” to CD8+ cytolytic T cells

  • Signal 1 - TCR-MHC interaction

  • Signal 2 - enhance binding of variety of T cell surface molecules to their ligands on APCs

  • TCR αβ + CD3 ITAMs phosphorylated by Lck/Fyn ➜ ZAP-70 ➜ LAT/SLP-76 nucleation ➜ PLCγ1, Ras/MAPK, PKCθ, calcineurin–NF-AT.

  • Immunologic synapse (c-SMAC, p-SMAC, d-SMAC) organizes signaling; mechanotransduction via TCR–pMHC under shear (Fig 23-14).

  • Costimulation

    • Signal 2 needed to avoid anergy: CD28–CD80/86 (activation), CTLA-4 (inhibition), ICOS–ICOSL (B-cell help), PD-1–PD-L1/2 (tolerance).

    • TNFR family: CD40–CD40L essential for B CSR; OX40, 4-1BB, CD27 modulate survival.

  • TCR signaling via CD3 dimers - evokes T cell lineage commitment and repertoire selection during development, maintains the peripheral T cell pool and further differentiates naive T cells into effector or memory cell populations upon immune stimulation

Peripheral T cell Maturation

Hallmark of adaptive immunity - generation of an antigen-specific response that can be rapidly recalled upon reexposure to the same antigen at a later time

  • CD8+ T cell - kills targets bearing cognate antigen; suppressor cells

  • CD4+ T cell - produce polarized sets of effector cytokines;

T cell activation - upregulation of CD2, CD58, LFA-1, CD29, and CD44 and increase the avidity of the interaction between the T cell and the antigen-bearing APC; CD45RA expressed on naive T cells is downregulated and replaced by CD45RO isoform

  • Expression of CD62L (L-selectin) and CCR7

  • CCR7 - lymph node-homing receptor that interacts with its ligand SLC on endothelial cells

    • Effector memory (CCR7-) - rapidly release cytokines and kill targets

    • Central memory (CCR7+) - interactions with dendritic cells and B cells orchestrate effective T cell help and secondary proliferation

Helper T cell Differentiation and function

  • Helper subsets determined by cytokine milieu & transcription factors (Fig 23-17)

    • TH1: IL-12+IFN-γ/T-bet ➜ IL-12/IFN-γ/TNF-α - Cell mediated immunity: Delayed Hypersensitivity (Infection)

    • TH2: IL-4/GATA-3 ➜ IL-4/IL-5/IL-13. - Humoral Immunity: Immunity to parasites (allergy)

    • TH17: TGF-β + IL-6/RORγt ➜ IL-17, IL-22. - Mucosal immunity: Protection at mucosal sites (inflammatory disease)

    • Treg: TGF-β + IL-2/FOXP3 ➜ immunosuppression. - Immune regulation: Suppression of effector cells (autoimmunity)

    • TFH: IL-6/IL-21 + BCL6 ➜ IL-21, CXCR5. - Humoral Immunity: Activation and differentiation of B cells to antibody screening cells (humoral immunodeficiency)

Cytotoxic T cell Differentiation and Function

Effector CD8+ - primary function is direct killing of APCs infected by virus or other intracellular organism;

  • cytotoxicity is antigen dependent, contact dependent and does not result in destruction of the cytotoxic T cell itself

  • release contents of cytotoxic granules into the immune synapse after contact an infected cell

    • Main components:

      • perforin - mediate delivery of granzymes into the cytoplasm; results in congenital HLH (deficiencies in CD8 and NK cytotoxic function)

      • serine esterases granzyme A and granzyme B

CD95-CD95L Pathway - induction of caspase-mediated apoptosis by engagement of CD95 (Fas, Apo-1) on target cells with its ligand CD95L (Fas ligand, CD178) on effector CD8+ T cells; upregulated on activation of T cells and NK cells;control of lymphoproliferation

  • mutations on CD95 does not cause overwhelming infection but generalized lymphoproliferation and autoimmunity in humans - ALPS

Control of Immune response

  • Autoimmunity - failure of self tolerance

    • Central or clonal deletion - deletion by negative selection in the thymus

    • Anergy - encounters antigen in the absence of costimulation; inability to proliferate to a specific antigen when rechallenged while maintaining the ability to proliferate in response to cytokine

    • Activation-induced cell death (AICD)- ensures that the robust expansion of antigen-specific T cells is short lived and contracted

    • Cell-mediated suppression or regulation

Nonconventional Lymphocytes

  • Subset of T cells that bear αβ TCR but recognize lipid antigens rather than peptide antigens

  • CD1-restricted T cells - invariant NK T cells bearing Vα24-Jα18 rearranged TCRα protein

    • Develop on the thymus from DP stage of development

    • Express CD1d rather than by thymic stroma

    • Recognize lipid presented by CD1

    • α-galactosylceramide - derived from marine sponge, highly potent agonist

    • Produce cytokines IL-4 and IFN-γ, more rapidly than conventional TCR αβ T cells - bridge between innate and adaptive immunity in diverse processes, including response to infection, antitumor immunity, autoimmune disease, and asthma

    • defects sickle-cell crisis pathogenesis.

  • TCR γδ T cells – enriched in gut epithelium;

    • Most do not express either the CD4 and CD8 coreceptor

    • recognize non-peptidic ligands and mycobacterial antigens

  • NK development: bone marrow & secondary lymphoid tissue; receptors (KIR, LIR) regulate missing-self recognition

    • Distinct subset of lymphocytes that do not rearrange the T cell antigen receptor genes

    • Capacity mediate spontaneous lysis of sensitive target cells, including certain tumor cells and hematopoietic cells to mediate antibody dependent cellular cytotoxicity (ADCC)

    • Role in allograft rejection and tumor surveillance

    • Derived from common lymphoid precursor cell or directly from precursors in the bone marrow or may transit through the thymus

Clinical Correlates & Molecular Defects

  • SCID phenotypes (Table 24-2): impact of ADA, PNP, RAG, Artemis, CD3, IL-7R, γc/JAK3, AK2.

  • Agammaglobulinemia: BTK, μ heavy chain, surrogate light chain defects.

  • Hyper-IgM: CD40L, CD40, AID, UNG.

  • Autoimmunity due to impaired negative selection (AIRE) or peripheral regulation (FOXP3, CTLA-4, IL-2Rα, STAT5b).

  • Cytokine receptor/shared JAK-STAT defects: γc/JAK3 (multiple cytokines), IL-21R (B help), IL-17RA/F (CMC).

  • NF-κB pathway (NEMO, IκBα) – ectodermal dysplasia + immunodeficiency.

  • Complement component & regulatory protein deficiencies ➜ encapsulated bacteria, SLE-like syndromes, hereditary angioedema.

Therapeutic Highlights

  • HSCT: curative for SCID, WAS, CD40L deficiency, HLH.

  • Gene therapy successes: ADA-SCID (retroviral & lentiviral vectors), γc-SCID (insertional oncogenesis lessons).

  • Biologics: CTLA4-Ig (abatacept) for autoimmunity; anti-BAFF (belimumab) for SLE; cytokine blockade (tocilizumab, etanercept, etc.).

  • IgG replacement (IVIG/SCIG) cornerstone for humoral PIDD; antibiotic prophylaxis adjunct.

  • Targeted treatments: eculizumab (anti-C5) for complement-mediated HUS; kallikrein inhibitors & C1-INH concentrates for hereditary angioedema.


Ethical, Evolutionary & Translational Insights
  • MHC polymorphism ensures species survival vs. rapidly mutating pathogens; but predisposes to drug hypersensitivity.

  • Somatic gene rearrangement & SHM exemplify programmed genomic instability yielding diversity but requiring robust DNA repair (implicated in cancer & immunodeficiency).

  • Newborn TREC screening saves lives through early HSCT for SCID – public-health paradigm.

  • CAR-T therapy leverages TCR signaling knowledge for cancer immunotherapy; safety depends on co-stimulatory domain engineering.

  • AIRE & FOXP3 mutations illuminate checkpoints of self-tolerance, guiding therapies for autoimmunity and transplant tolerance.

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