Immunodeficiencies & Monoclonal Antibody Technology

Practice flashcards covering innate and adaptive immunodeficiencies, HIV pathogenesis and treatment targets, and monoclonal antibody production technologies based on PHAR 641 lecture materials.

Chronic Granulomatous Disease

A condition characterized by impaired Reactive Oxygen Species ($ROS$) production in phagocytes, leading to increased bacterial and fungal infection.

Leukocyte Adhesion Deficiency

An immunodeficiency resulting in impaired leukocyte adhesion to endothelium and tissue migration, which increases susceptibility to bacterial and fungal infections.

Chediak-Higashi Syndrome

A disorder involving impaired vesicle fusion and lysosomal function in neutrophils, macrophages, and dendritic cells, causing recurrent bacterial infections.

Toll-like Receptor Defects

Defects in $TLR$ signaling pathways that diminish transcription factor $NF-\text{kB}$ activity, weakening the inflammatory response to microorganisms.

Congenital (Primary) Immunodeficiencies

Genetic defects present at birth that affect lymphocyte maturation or function, the most severe being Severe Combined Immunodeficiencies ($SCID$).

Acquired (Secondary) Immunodeficiencies

Deficiencies resulting from external factors such as chemotherapy, irradiation, immunosuppressive drugs, malnutrition, splenectomy, or infections like $HIV$.

Severe Combined Immunodeficiencies (SCID)

Lethal immune function failures involving defects in both $T$ and $B$ cells that require bone marrow transplantation or reconstitution.

Common $\text{gamma}$ Chain ($gc$) Mutation

The most common cause of $SCID$ (\text{at least } 50 \text{%}) leading to a lack of $T$ cells due to a cytokine receptor defect affecting $IL-7$ signaling.

Recombination-Activating Gene (RAG) Mutation

A genetic defect causing defective maturation of lymphocytes, resulting in decreased $T$ and $B$ cells and reduced $Ig$.

Bruton's Tyrosine Kinase (BTK) Mutation

A mutation that blocks $B$ cell maturation, leading to Agammaglobulinemia, characterized by a decrease in all $Ig$ isotypes and reduced $B$ cell numbers.

X-Linked Hyper-IgM Syndrome

A lymphocyte function defect caused by mutations in $CD40L$, resulting in decreased Cell-Mediated Immunity ($CMI$) and decreased class switching.

Autosomal Hyper-IgM Syndrome

A condition caused by mutations in $AID$ where there is no class switching (only $IgM$ produced) despite normal $T$ cell function and $CMI$.

CD4+ Cell Count

The primary biomarker used to assess $HIV$ treatment outcomes because the virus specifically infects and progressively impairs these cells.

HIV Therapeutic Targets

The three strategic points of the viral life cycle targeted by drugs: Reverse Transcriptase Inhibitors, Integrase Inhibitors, and Protease Inhibitors.

Hybridoma Technique

A method for monoclonal antibody production developed in $1975$ by Milstein and Köhler.

Phage Display

A companion technology for monoclonal antibody production used to screen for antibody binding within a “Naïve” Human Antibody Library.

Biosimilars

Proprietary biologic drugs “Highly Similar” to an $FDA$-approved reference product, distinguished by a mandatory $4$ letter suffix.