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Multiple Choice
What factors must be considered when choosing a vector for a gene therapy trial?
A
The vector's ability to integrate into the host genome
B
The vector's ability to replicate independently
C
The vector's ability to target specific cells
D
The vector's ability to cause an immune response
Verified step by step guidance
1
Understand the purpose of a vector in gene therapy: A vector is used to deliver genetic material into cells. It is crucial to choose a vector that can efficiently and safely deliver the therapeutic gene to the target cells.
Consider the vector's ability to integrate into the host genome: Some vectors, like retroviruses, integrate their genetic material into the host genome, which can provide long-term expression of the therapeutic gene. However, this can also pose risks such as insertional mutagenesis.
Evaluate the vector's ability to replicate independently: Vectors that replicate independently, such as plasmids, may not integrate into the host genome, reducing the risk of insertional mutagenesis but potentially requiring repeated administrations.
Assess the vector's ability to target specific cells: It is important for the vector to have specificity for the target cells to ensure that the therapeutic gene is delivered to the correct location, minimizing off-target effects and maximizing therapeutic efficacy.
Examine the vector's potential to cause an immune response: The immune system may recognize the vector as foreign and mount an immune response, which can reduce the effectiveness of the therapy and pose safety concerns. Choosing a vector with low immunogenicity is crucial for successful gene therapy.