Physiology, Biochemistry, Drug Delivery, Gene Therapy, Immunotherapy, and Biosensors

Vocabulary flashcards covering topics in physiology, biochemistry, routes of drug administration, gene therapy methods, adaptive and innate immunity, antibody therapeutics, and biosensor mechanics.

Small Molecule

A chemically synthesized, low molecular weight therapeutic drug. Examples include Aspirin, penicillin, and ibuprofen.

Nucleic Acid (Drug)

Polymers of nucleotides used as therapeutics, operating via the pathway: DNA (in nucleus; transcription) to RNA (in cytoplasm; copy of DNA; translation) to protein.

Peptide

A short protein consisting of a string of amino acids ($< 100$, $< 50$). Examples include Ozempic and Wegovy.

Protein (Drug)

A macromolecule composed of amino acids ($20$ natural amino acids, each with a 3-letter and 1-letter code) that feature a variable group (R) causing variation between them.

Amino Acid Structure

The structural organization of an amino acid featuring a central carbon bonded to a hydrogen atom (H), an Amino Group, a Carboxyl Group, and a variable side chain (R).

Epithelium

A type of biological barrier forming the linings of organs and blood vessels, functioning structurally like brick and mortar.

Phagocytosis

A form of endocytosis known as cellular eating, in which immune cells digest large molecules and microorganisms.

Mononuclear Phagocyte System (MPS)

A system of specialized cells that removes old cells and foreign objects from circulation, leading to phagocytosis.

Opsonin

Any substance that enhances phagocytosis by preparing foreign material for ingestion ('preparing the feast').

Complement System

A system of proteins that pokes holes in cell membranes, resulting in cell death.

Pinocytosis

A form of endocytosis characterized as cellular drinking.

Receptor-Mediated Endocytosis

An endocytic pathway (sometimes referred to as Clathrin-mediated) in which a ligand binds to a cell surface receptor to trigger internalization, after which lysosomes degrade low pH enzymes.

Routes of Drug Administration Table

A summary table outlining administration routes (Intramuscular, Nasal, Intravenous, Oral, Topical, Opthalmic) along with drug examples, advantages, and disadvantages.

Intramuscular Administration

A route of drug administration (e.g., vaccine, insulin) that is faster acting and offers high bioavailability, but can cause discomfort.

Nasal Administration

A drug administration route (e.g., Flonase) that acts quickly and crosses the blood-brain barrier (BBB) more easily, but is short-lived.

Intravenous Administration

A drug route (e.g., propofol for anesthesia) that provides high potency and quick action, but requires a medical professional, is expensive, and causes discomfort.

Oral Administration

A common drug administration route (e.g., ibuprofen) favored for ease and patient compliance, but limited by limited absorption and drug degradation.

Topical Administration

A self-administered route (e.g., hydrocortizone) with limited risk of infection, but restricted to specific applications and capable of causing a rash.

Opthalmic Administration

A localized route (e.g., eye drops) providing high potency, but associated with high user error, discomfort, and frequent administration requirements.

Therapeutic Window

The target drug concentration range lying above the minimum effective concentration (MEC) and below the minimum safe concentration (MSC).

Plasma Concentration vs. Time Profile

A pharmacokinetic plot showing plasma drug concentration over time across administration routes, highlighting peak concentration ($C_{\text{max}}$) and time to peak ($t_{\text{max}}$).

Nanocarriers

Delivery constructs that improve drug solubility and stability, aiding in crossing cell membranes, targeted delivery, triggered release, and co-encapsulation.

Gene Therapy

The transfer of nucleic acids (DNA/RNA) into a patient to achieve a therapeutic effect.

Polyethylene Glycol (PEG)

A chemical linker used in gene delivery formulations to prevent uptake by phagocytic cells.

Somatic Cells

All non-reproductive body cells, which are targeted in ethical gene therapy to prevent inherited genetic alterations in offspring.

Germline Cells

Reproductive cells that are not considered ethical to edit due to permanent effects passed to future generations.

Gene Augmentation

A gene therapy strategy that increases gene expression by introducing a wild-type (WT) non-mutated gene to treat a loss-of-function mutation.

Gene Suppression

A gene therapy strategy that decreases gene expression to block the harmful effects of a gain-of-function mutation.

Genome Editing

Directly changing the existing natural DNA sequences within a cell, such as through CRISPR.

SiRNA (Short Interfering RNA)

Double-stranded RNA complementary to a targeted gene, utilized for gene suppression or silencing.

ASOs (AntiSense Oligonucleotides)

Single-stranded DNA complementary to a targeted gene, utilized for gene suppression or silencing.

CRISPR

Clustered regularly interspaced palindromic repeats; a bacterial defense system against phages (bacterial viruses) repurposed for precise genome editing.

Physical gene transfer method

refers to techniques used to introduce genetic material directly into cells, often employing methods such as electroporation, microinjection, or gene guns.

viral gene transfer method

a method that utilizes viral vectors to deliver genetic material into host cells, exploiting the natural ability of viruses to infect cells and integrate their genetic material.

Nonviral vectors gene transfer methods

Liposomes, polymers, nanoparticles 

Immunotherapy

A disease treatment approach that harnesses or modulates the body's immune system.

Innate vs. Adaptive Immunity Table

A comparative reference chart comparing speed, specificity, and primary cellular/molecular components of innate and adaptive immune systems.

Innate Immunity

An immediate, non-specific immune system acting within hours, consisting of macrophages, mast cells, neutrophils, NK cells, physical barriers (skin, mucus membranes), and the complement system.

Adaptive Immunity

A highly specific immune response that develops within days or weeks, composed primarily of B cells, antibodies, and T cells.

Macrophages

Phagocytic cells present in every organ that search for pathogens, infected cells, dead cells, and debris; they adopt M1 (inflammatory) or M2 (anti-inflammatory) phenotypes based on signals.

Natural Killer Cells (NK Cells)

A small fraction of white blood cells (WBCs) that destroy infected or cancer cells, characterized by a short lifespan that makes them challenging to work with.

Cytotoxic T Cells

T lymphocytes maturing in the thymus that recognize MCH-I to directly kill infected cells and tumor cells.

Helper T-Cells

T lymphocytes maturing in the thymus that recognize MCH-II and send signals to activate and coordinate other immune cells.

B Cells

Bone marrow-derived lymphocytes that produce antibodies against antigens; includes antibody-releasing Plasma B Cells and antigen-remembering Memory B Cells.

Blocking antigen action

Prevent something from doing its normal harmful action.

Example: Humira

Blocks TNF → prevents inflammatory cascade.

Neutralizing

Bind to a harmful molecule/toxin so it can't act.

Example: Zinplava

Binds toxin produced by C. difficile.

Recruiting

Antibody binds to a target cell, and immune cells recognize the antibody and destroy/engulf the target.

Example: Trastuzumab

Binds HER2 on some breast cancer cells.

Delivering

Use antibody specificity to deliver a drug to a target.

Example:

Adcetris = antibody-drug conjugate (ADC)

Binds CD30 on lymphoma cells.

CAR T-Cell Therapy

A cellular immunotherapy where T-cells are harvested from patient blood, genetically engineered into CAR T-cells, expanded to millions in the laboratory, and re-infused into the patient to destroy cancer.

Biosensor

A device that detects a specific biomolecule (analyte) and converts its presence into a quantifiable and displayable signal.

Polymerase Chain Reaction (PCR)

A molecular technique used to amplify a small amount of DNA into a large quantity of DNA.

Diagnostic Test Results Table

A 2x2 contingency matrix comparing actual disease status against test results to evaluate true positives, false positives, false negatives, and true negatives.

Sensitivity

The probability of obtaining a positive test result when the patient actually has the disease, calculated as $\text{Sensitivity} = \frac{\text{TP}}{\text{Tdisease}} = \frac{10}{15} \thickapprox 0.667$.

$\text{Specificity} = \frac{\text{TM}}{\text{Tnondisease}} = \frac{45}{85} \thickapprox 0.529$.

The probability of obtaining a negative test result when the patient does not have the disease, calculated as