Gene Therapy: A Comprehensive Guide for Medical Students

Question 1

Which option is NOT a type of gene therapy?

Accepted Answer

Palliative therapy

Answer

Gene replacement therapy

Answer

Gene silencing

Answer

Gene editing

Question 2

In the context of gene therapy for inherited diseases, which ethical concern is particularly relevant?

Accepted Answer

Germline editing

Answer

Off-target effects

Answer

Unequal access to treatment

Question 3

Which subtype of gene therapy involves replacing a defective gene with a functional one?

Accepted Answer

Gene replacement therapy

Answer

Gene editing

Answer

Gene silencing

Answer

Somatic gene therapy

Question 4

Which type of gene therapy targets non-germ cells?

Accepted Answer

Somatic gene therapy

Answer

Germline gene therapy

Answer

Systemic gene therapy

Answer

Targeted gene therapy

Question 5

Which technique is NOT considered a major type of gene therapy?

Accepted Answer

Gene augmentation therapy

Answer

Gene replacement therapy

Answer

Gene editing therapy

Answer

Gene silencing therapy

Question 6

Which method is used to add a new gene into a cell?

Accepted Answer

DNA transfection

Answer

Genome editing

Answer

RNA interference

Answer

CRISPR-Cas9

Question 7

Which organization is responsible for regulating gene therapy in the United Kingdom?

Accepted Answer

Medicines and Healthcare products Regulatory Agency (MHRA)

Answer

Human Fertilisation and Embryology Authority (HFEA)

Answer

National Institute for Health and Care Excellence (NICE)

Answer

British Medical Association (BMA)

Question 8

What's the key distinction between in vivo and ex vivo gene therapy?

Accepted Answer

In vivo therapy modifies genes within the body, while ex vivo therapy modifies genes outside the body.

Answer

In vivo therapy uses viral vectors, while ex vivo therapy uses non-viral vectors.

Answer

In vivo therapy is more effective than ex vivo therapy.

Answer

Ex vivo therapy is safer than in vivo therapy.

Question 9

What is gene editing's role in gene therapy?

Accepted Answer

Correcting genetic defects by altering specific DNA sequences.

Answer

Inserting new genes into the genome.

Answer

Silencing disease-causing genes.

Answer

Regulating gene expression.

Question 10

What's the difference between somatic and germline gene editing?

Accepted Answer

Somatic gene editing affects only the recipient, while germline gene editing affects both the individual and their descendants.

Answer

Somatic gene editing is more precise than germline gene editing.

Answer

Germline gene editing is more effective than somatic gene editing.

Answer

Somatic and germline gene editing are identical.

Question 11

How could gene therapy potentially be used to treat genetic disorders? Explain using cystic fibrosis as an example.

Accepted Answer

Replace the faulty CFTR gene with a healthy copy.

Answer

Gene therapy is not yet a viable treatment for cystic fibrosis.

Answer

Gene therapy is only effective for recessive genetic disorders like cystic fibrosis.

Answer

Gene therapy for cystic fibrosis is completely safe.

Question 12

What is the key difference between somatic and germline gene editing approaches, and how do these differences impact the individuals affected?

Accepted Answer

Somatic gene editing affects only the individual receiving treatment, while germline gene editing can have effects on future generations.

Answer

Germline gene editing is more efficient than somatic gene editing.

Answer

Somatic gene editing is used only for treating genetic disorders, while germline gene editing is used for human enhancement.

Question 13

What is the fundamental principle behind gene replacement therapy?

Accepted Answer

Inserting a functional gene to correct a genetic defect

Answer

Editing or modifying an existing gene

Answer

Blocking the expression of a harmful gene

Answer

Combining gene therapy with other treatments

Question 14

Which gene editing technique utilizes the CRISPR-Cas system to introduce precise alterations in DNA?

Accepted Answer

CRISPR-Cas

Answer

Gene silencing

Answer

Gene addition

Answer

Genome editing

Question 15

What is the purpose of gene silencing in gene therapy?

Accepted Answer

Suppressing the expression of harmful genes

Answer

Correcting genetic mutations

Answer

Enhancing gene expression levels

Answer

Replacing defective genes

Question 16

Which of the following is not a type of gene therapy?

Accepted Answer

Genetic counseling

Answer

Gene replacement therapy

Answer

Gene editing

Answer

Gene silencing

Question 17

Which of the following is NOT a recognized approach in gene therapy?

Accepted Answer

Virotherapy

Answer

Gene replacement therapy

Answer

Gene silencing

Answer

Gene editing

Question 18

What is the primary aim of gene replacement therapy?

Accepted Answer

To introduce a functional gene to replace a defective or missing gene.

Answer

To modify a mutated gene.

Answer

To suppress the expression of a specific gene.

Question 19

Which of the following is a potential complication associated with gene therapy?

Accepted Answer

Immune response to the viral vector

Answer

Reduced healthcare costs

Answer

Improved patient health

Answer

Elimination of genetic diseases

Question 20

How does the CRISPR-Cas9 gene editing system work?

Accepted Answer

It uses a guide RNA to direct a nuclease to a specific DNA sequence for modification.

Answer

It replaces a defective gene with a functional one.

Answer

It inhibits gene expression by binding to mRNA.

Question 21

Which gene therapy approach utilizes siRNA?

Accepted Answer

Gene silencing

Answer

Gene editing

Answer

Viral vector therapy

Answer

Gene replacement therapy

Question 22

What ethical concern is most prominent when considering gene therapy in germline cells?

Accepted Answer

The potential impact on future generations

Answer

The informed consent of participants

Answer

The cost-effectiveness of the therapy

Answer

The availability of alternative treatments

Question 23

What is the primary function of viral vectors in gene therapy?

Accepted Answer

To deliver therapeutic genes into target cells

Answer

To silence the expression of disease-causing genes

Answer

To stimulate the immune system against cancer cells

Question 24

What is a current limitation of gene therapy techniques?

Accepted Answer

Off-target effects

Answer

Affordable costs

Answer

High success rates

Answer

Applicability to all genetic diseases

Question 25

What is the likely future direction of gene therapy research?

Accepted Answer

Continued advancements in gene editing and delivery methods

Answer

Replacement by traditional therapies

Answer

Limited applicability to common diseases