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Question 1

What is the primary cause of hemophilia?

Answer 1

Mutations in clotting enzymes, such as Factor VIII (Hemophilia A) or Factor IX (Hemophilia B).

Question 2

What was a major breakthrough in hemophilia treatment?

Answer 2

The development of recombinant Factor VIII, reducing reliance on blood donations and eliminating viral contamination risks.

Question 3

What is the significance of sensitivity and specificity in diagnostics?

Answer 3

Sensitivity measures a test’s ability to identify true positives, while specificity measures its ability to identify true negatives.

Question 4

How does personalized medicine improve drug efficacy?

Answer 4

By tailoring treatments based on genetic, phenotypic, and molecular profiles, reducing side effects and improving outcomes.

Question 5

What is a bispecific antibody?

Answer 5

An engineered antibody that binds to two different targets, improving therapeutic effects in diseases like cancer.

Question 6

What is a fusion protein in biologics?

Answer 6

A protein engineered by combining two or more distinct proteins to enhance drug stability or targeting.

Question 7

What is the function of cytochrome P450 enzymes?

Answer 7

These enzymes metabolize drugs in the liver, playing a major role in drug detoxification and clearance.

Question 8

Why does drug metabolism vary between individuals?

Answer 8

Factors such as genetics, age, diet, disease state, and drug-drug interactions affect metabolism rates.

Question 9

What is drug half-life?

Answer 9

The time it takes for the concentration of a drug in the bloodstream to be reduced by 50%.

Question 10

What is the significance of the area under the curve (AUC) in pharmacokinetics?

Answer 10

AUC represents the total drug exposure over time, helping to assess bioavailability and dosing strategies.

Question 11

How can gut bacteria impact drug efficacy?

Answer 11

Gut microbes can metabolize drugs into active or inactive forms, altering their effects and toxicity.

Question 12

What is an example of a microbiome-derived drug?

Answer 12

Fecal microbiota transplant (FMT) for treating Clostridioides difficile infections.

Question 13

What is homologation in drug design?

Answer 13

A method where a chemical chain is extended to modify drug properties such as solubility and binding affinity.

Question 14

What is the purpose of prodrug design?

Answer 14

Prodrugs are inactive forms of drugs that become active after metabolism, improving stability, absorption, or targeting.

Question 15

What is the importance of polar surface area (PSA) in drug design?

Answer 15

PSA affects a drug’s ability to cross biological membranes and its oral bioavailability.

Question 16

What is the significance of molecular flexibility in drug development?

Answer 16

High flexibility can reduce target binding specificity, while rigid molecules may improve selectivity and potency.

Question 17

What is the role of dendritic cells in cancer immunotherapy?

Answer 17

Dendritic cells present antigens to T cells, helping to activate the immune response against tumors.

Question 18

What is an oncolytic virus?

Answer 18

A genetically modified virus that selectively infects and destroys cancer cells while sparing healthy tissue.

Question 19

What is an example of an FDA-approved CAR-T therapy?

Answer 19

Tisagenlecleucel (Kymriah), used for B-cell acute lymphoblastic leukemia (ALL).

Question 20

What is the mechanism of immune checkpoint inhibitors?

Answer 20

They block inhibitory receptors like PD-1 and CTLA-4 on T cells, enhancing immune system activity against cancer.

Question 21

Which of the following best describes the primary action of anti-PD-1 or anti-CTLA-4 immunotherapy?

Answer 21

(b) Blockade of inhibitory signaling from the cancer cell, thus restoring T-cell activation.

Question 22

Which of the following immune cells are primarily responsible for killing tumor cells in response to checkpoint inhibitor therapy?

Answer 22

(a) Cytotoxic T lymphocytes (CTLs).

Question 23

What is the main purpose of CAR-T cell therapy?

Answer 23

(c) Engineering patient T cells to recognize and attack cancer cells.

Question 24

Which of the following is an example of a monoclonal antibody therapy targeting HER2-positive breast cancer?

Answer 24

(d) Trastuzumab (Herceptin).

Question 25

Which phase of metabolism is primarily responsible for making drugs more hydrophilic for excretion?

Answer 25

(a) Phase I reactions (e.g., oxidation, reduction, hydrolysis).

Question 26

Which enzyme family is responsible for the majority of drug metabolism in the liver?

Answer 26

(b) Cytochrome P450 enzymes (CYPs).

Question 27

A patient has a genetic polymorphism leading to reduced CYP2D6 activity. How might this affect drug metabolism?

Answer 27

(c) Decreased metabolism of CYP2D6 substrates, leading to increased drug levels and potential toxicity.

Question 28

What is the main determinant of a drug’s oral bioavailability?

Answer 28

(d) First-pass metabolism and intestinal absorption.

Question 29

A drug with a high volume of distribution (Vd) is most likely to:

Answer 29

(b) Distribute extensively into tissues rather than remaining in the plasma.

Question 30

Which PK parameter is most directly related to the duration of drug action?

Answer 30

(c) Half-life (t1/2).

Question 31

How can the gut microbiome alter drug metabolism?

Answer 31

(a) Bacteria can metabolize drugs into active or inactive forms, affecting efficacy and toxicity.

Question 32

Which of the following is an example of microbiome-mediated drug metabolism?

Answer 32

(b) The conversion of L-dopa into dopamine by gut bacteria.

Question 33

What is the goal of lead optimization in drug discovery?

Answer 33

(d) Improving potency, selectivity, and pharmacokinetics while minimizing toxicity.

Question 34

Which of the following is an example of a bioisostere used to enhance drug properties?

Answer 34

(c) Replacing a carboxylic acid (-COOH) with a tetrazole to improve metabolic stability.

Question 35

Which condition is most commonly treated with enzyme replacement therapy (ERT)?

Answer 35

(a) Lysosomal storage disorders such as Gaucher’s disease.

Question 36

Which of the following is a major limitation of enzyme replacement therapy?

Answer 36

(d) The need for frequent infusions and potential immune responses.

Question 37

Which of the following is an example of a companion diagnostic?

Answer 37

(b) HER2 testing to determine eligibility for trastuzumab therapy.

Question 38

What is the primary benefit of companion diagnostics in oncology?

Answer 38

(c) Identifying patients who are most likely to benefit from targeted therapy.

Question 39

Which of the following best describes pharmacogenomics?

Answer 39

(a) The study of how genetic differences affect drug response.

Question 40

Which physicochemical property is most important for predicting oral bioavailability?

Answer 40

Lipophilicity (logP and logD), as it affects membrane permeability and solubility.

Question 41

What is Lipinski’s Rule of 5 used for?

Answer 41

Predicting drug-likeness and oral bioavailability based on molecular weight, hydrogen bonding, and lipophilicity.

Question 42

Which property is considered in both Lipinski’s and Veber’s rules?

Answer 42

Molecular flexibility, measured by the number of rotatable bonds.

Question 43

What is the role of X-ray crystallography in drug discovery?

Answer 43

Determining the 3D structure of proteins to enable structure-based drug design (SBDD).

Question 44

Why is computational docking useful in drug discovery?

Answer 44

It allows rapid screening of virtual compound libraries against protein targets to identify potential inhibitors.

Question 45

Which database is most commonly used to store protein structures?

Answer 45

The Protein Data Bank (PDB).

Question 46

Which of the following best describes high-throughput screening (HTS)?

Answer 46

Rapidly testing thousands to millions of compounds for biological activity in an automated process.

Question 47

What is the purpose of hit confirmation in drug discovery?

Answer 47

To verify the biological activity of initial hits and eliminate false positives.

Question 48

Which statistical measure is used to assess screening quality?

Answer 48

Z’ factor, which evaluates assay robustness based on signal-to-background variation.

Question 49

Which of the following is an example of an ‘undruggable’ target?

Answer 49

Transcription factors, as they lack traditional small-molecule binding pockets.

Question 50

What is the mechanism of action of PROTACs?

Answer 50

They induce targeted protein degradation by recruiting an E3 ligase to ubiquitinate the target protein.

Question 51

What are molecular glues?

Answer 51

Small molecules that stabilize protein-protein interactions to trigger target degradation.

Question 52

What is network pharmacology?

Answer 52

A systems biology approach that considers the interactions of drugs with multiple targets rather than a single target.

Question 53

Why is multi-target drug design beneficial?

Answer 53

It can enhance efficacy and reduce resistance by modulating multiple pathways simultaneously.

Question 54

Which class of receptors is targeted by the largest number of FDA-approved drugs?

Answer 54

G protein-coupled receptors (GPCRs), which regulate key physiological functions.

Question 55

What is the difference between orthosteric and allosteric ligands?

Answer 55

Orthosteric ligands bind to the active site, while allosteric ligands modulate receptor activity by binding elsewhere.

Question 56

What is the mechanism of action of HIV protease inhibitors?

Answer 56

They prevent the cleavage of viral polyproteins, blocking the maturation of infectious virus particles.

Question 57

Why was the development of Paxlovid important for COVID-19 treatment?

Answer 57

Paxlovid (nirmatrelvir/ritonavir) targets the SARS-CoV-2 main protease, preventing viral replication.

Question 58

Which omics approach is most useful for identifying drug targets based on gene expression?

Answer 58

Transcriptomics, as it profiles RNA expression levels across different conditions.

Question 59

How does pharmacogenomics influence drug therapy?

Answer 59

It tailors drug selection and dosing based on an individual’s genetic profile to maximize efficacy and minimize side effects.

Question 60

What is the function of ion channels in drug targets?

Answer 60

They regulate the movement of ions across cell membranes, influencing excitability and signaling.

Question 61

Which drug class targets voltage-gated sodium channels?

Answer 61

Local anesthetics, such as lidocaine, which block nerve conduction to prevent pain transmission.