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NEET Questions / Botany / Biotechnology and Its Applications / Gene Therapy
A severe combined immunodeficiency (SCID) patient undergoes gene therapy using a retroviral vector to deliver the functional ADA gene. A potential long-term complication specifically associated with this vector type is:
Insertional mutagenesis leading to oncogenesis
Hypersensitivity reaction to the viral vector
Development of neutralizing antibodies against ADA
Rapid clearance of the vector by the immune system
Which of the following is NOT a crucial consideration when designing a CRISPR-Cas9 system for gene therapy targeting a specific genetic disorder?
Ensuring precise targeting of the Cas9 enzyme to the mutated gene
Minimizing off-target effects of the Cas9 enzyme
Choosing an appropriate delivery system for the CRISPR-Cas9 components
Enhancing the host's immune response to the Cas9 enzyme
In gene therapy for cystic fibrosis, the most significant barrier to successful gene transfer to the lung epithelium is:
The rapid turnover rate of lung epithelial cells
The thick mucus layer hindering vector access to target cells
The lack of suitable viral vectors for lung tissue
The immune system's rapid clearance of the therapeutic gene
Suicide gene therapy for cancer utilizes genes that convert a prodrug into a cytotoxic agent. Which combination is most likely to be used in this approach?
Adenosine deaminase (ADA) gene and polyethylene glycol (PEG)
Herpes simplex virus thymidine kinase (HSV-TK) gene and ganciclovir
Dystrophin gene and corticosteroids
Factor VIII gene and desmopressin
Ex vivo gene therapy involves:
Directly injecting the therapeutic gene into the patient
Modifying cells outside the body and then reintroducing them
Using a viral vector to deliver the gene in vivo
Modifying germline cells to correct genetic defects
Zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR-Cas9 are all tools used in gene editing. What is a key advantage of CRISPR-Cas9 over ZFNs and TALENs?
Higher target specificity and lower off-target effects
Easier design and implementation due to RNA-guided targeting
Ability to target multiple genes simultaneously without modification
Greater stability and longer lifespan within the target cell
Which of the following is the primary goal of gene therapy?
To enhance athletic performance
To treat or prevent disease by correcting genetic defects
To alter physical appearance
To create genetically modified organisms
What is a vector in the context of gene therapy?
A type of white blood cell
A disease-causing agent
A carrier used to deliver therapeutic genes into cells
A protein that repairs damaged DNA
Which type of diseases is gene therapy most commonly targeted towards?
Infectious diseases
Genetic disorders
Injuries
Nutritional deficiencies
What is the role of a 'functional gene' in gene therapy?
To cause the disease
To protect against infections
To replace a faulty gene
To enhance immune response
