Which gene therapy approach could potentially treat a recessive loss-of-function genetic disease by restoring missing protein function?
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Correct answer: Adding a functional copy of the defective gene
In loss-of-function recessive disorders, adding a correct functional copy of the gene restores protein production and corrects disease — a classic gene-addition application.
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More Gene therapy basics questions
- Which of the following is an advantage of using in vivo gene therapy over ex vivo gene therapy?
- Why is immune response a major concern in gene therapy using viral vectors?
- What is the primary goal of gene therapy?
- Which of the following is the most common type of delivery vector used in gene therapy?
- Which of the following describes an “in vivo” gene therapy approach?
- Which strategy describes “ex vivo” gene therapy?