The Question is from neet-botany biotechnology biotechnological-applications-in-medicine, and it was created by the author Mr. NARESH KUMAR K

Question

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?

Mr. NARESH KUMAR K · Accepted Answer

## CORE INFORMATION
✓ **Answer**: C
## CONCEPT FRAMEWORK
 **Primary Concept**: Gene therapy challenges and overcoming limitations
 **Related Topics**: Adeno-associated virus (AAV) vectors, neutralizing antibodies, immunogenicity, gene delivery systems
 **Prerequisites**: Understanding of gene therapy principles, immune response to viral vectors
## SOLUTION PATHWAY
**Given Information**:
  * Limited efficacy of AAV-based gene therapy for cystic fibrosis due to pre-existing neutralizing antibodies.
  * Neutralizing antibodies target the AAV capsid.
**Method & Steps**:
 1. Analyze the problem: The primary obstacle is the pre-existing immune response neutralizing the AAV vector before it can deliver the CFTR gene. 
  Reasoning: The body's immune system recognizes and destroys the AAV vector before it can reach the target cells.
  Formula/Rule: None directly applicable, this is a qualitative analysis problem.
 2. Evaluate the options:
  Working: Let's examine each option's effectiveness in addressing the pre-existing antibodies:
  A. Increasing the dosage might overwhelm the immune response in some individuals but is not a targeted solution and could lead to increased toxicity.
  B. Immunosuppressants broadly suppress the immune system, increasing the risk of infection and other complications. This is a risky approach.
  C. Altering the AAV capsid surface epitopes directly targets the problem by making the vector less recognizable to the pre-existing antibodies. This is a precise and targeted strategy.
  D. Switching vectors changes the problem, but doesn't solve the underlying issue of pre-existing immunity.  The patient might have pre-existing immunity to the new vector as well.
 3. Choose the best strategy: Engineering AAV capsids is the most effective and targeted approach to overcome pre-existing immunity.
  Result: Option C
  Verification: Option C directly addresses the root cause of the problem – the neutralizing antibodies targeting the AAV capsid – without introducing significant additional risks.
 ## OPTION ANALYSIS
A :x: Increasing the dosage is unlikely to overcome the problem of pre-existing neutralizing antibodies and could lead to increased toxicity.
B :x: Immunosuppression is a high-risk strategy with potential for severe side effects and is not a targeted approach.
C ✓: Engineering altered AAV capsids is a precise and effective strategy to evade the immune response and deliver the functional CFTR gene.
D :x: Switching to a different vector doesn't address the underlying issue of pre-existing neutralizing antibodies and might lead to similar problems with the new vector.
 ## LEARNING AIDS
 **Common Mistakes**:
  1. Choosing immunosuppression: This is a high-risk approach with many side effects.
  2. Focusing solely on increasing the dose: This doesn't address the root cause of the problem.
 **Quick Tips**:
  1. Identify the core problem: Pre-existing antibodies targeting the AAV capsid.
  2. Choose the most targeted solution: Engineering the AAV capsid to evade the immune response.

Mr. NARESH KUMAR K · Answer

Increasing the dosage of the AAV vector.

Mr. NARESH KUMAR K · Answer

Administering immunosuppressants prior to gene therapy.

Mr. NARESH KUMAR K · Answer

The correct Option is "Engineering AAV capsids with altered surface epitopes to evade pre-existing immunity."

Mr. NARESH KUMAR K · Answer

Switching to a lentiviral vector delivery system.

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