Prepare for NEET with Botany-specific practice questions. Covering all major Botany chapters, this is perfect for your NEET Biology needs.
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
A scientist is attempting to develop a novel gene therapy for cystic fibrosis. They are utilizing a modified adeno-associated virus (AAV) vector to deliver the functional CFTR gene. However, pre-clinical trials show limited efficacy due to pre-existing neutralizing antibodies against the AAV capsid in a significant portion of the target population. Which of the following strategies would be MOST effective in overcoming this limitation?
Increasing the dosage of the AAV vector.
Administering immunosuppressants prior to gene therapy.
Engineering AAV capsids with altered surface epitopes to evade pre-existing immunity.
Switching to a lentiviral vector delivery system.
A patient exhibits severe combined immunodeficiency (SCID) due to a deficiency in adenosine deaminase (ADA). Which gene therapy approach is MOST likely to provide a long-term solution?
In vivo gene therapy using a retroviral vector directly injected into the bone marrow.
Administering purified ADA enzyme intravenously.
Ex vivo gene therapy using autologous hematopoietic stem cells.
Bone marrow transplant from an HLA-matched sibling.
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
