Multiple Choice
What factors must be considered when choosing a vector for a gene therapy trial?
29. Heredity
Gene Therapy
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Why is gene therapy for Severe Combined Immunodeficiency (SCID) often performed as an ex vivo therapy?
A
It avoids the need for any surgical procedures.
B
It allows for direct modification of genes within the patient's body.
C
It enables the correction of genetic defects in cells outside the body before reintroduction.
D
It is less expensive and more efficient than in vivo methods.
Verified step by step guidance1
Understand the concept of ex vivo therapy: Ex vivo therapy involves removing cells from the patient's body, modifying them outside the body, and then reintroducing them. This is different from in vivo therapy, where the modification occurs directly within the body.
Recognize the challenges of in vivo gene therapy: In vivo methods require delivering genetic material directly to the target cells within the body, which can be complex and less controlled compared to ex vivo methods.
Identify the benefits of ex vivo therapy for SCID: By modifying cells outside the body, ex vivo therapy allows for precise control over the genetic modification process, ensuring that only the desired changes are made before the cells are returned to the patient.
Consider the safety and efficacy of ex vivo therapy: Ex vivo methods can reduce the risk of unintended effects, as the modified cells can be thoroughly tested for safety and efficacy before being reintroduced into the patient.
Conclude why ex vivo therapy is preferred for SCID: The ability to correct genetic defects in a controlled environment outside the body, ensuring safety and effectiveness, makes ex vivo therapy a suitable approach for treating SCID.
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