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In this example of gene therapy, the treatment is successful if the A - VCE - SSCE Biology - Question 9 - 2012 - Paper 1
Question 9
In this example of gene therapy, the treatment is successful if the A. normal allele for the gene is inserted into the nuclear DNA of airway cells. B. delivery parti... show full transcript
Worked Solution & Example Answer:In this example of gene therapy, the treatment is successful if the A - VCE - SSCE Biology - Question 9 - 2012 - Paper 1
Step 1
C. normal allele for the gene is expressed in the airway cells.
Answer
For gene therapy to be deemed successful, it is crucial that the introduced normal allele is actually expressed within the target airway cells. This expression means that the cells are not just receiving the gene but also producing the corresponding functional protein necessary for correcting the dysfunction caused by the gene defect. Without this expression, the therapy would not achieve its intended effect, even if the normal gene is present in the cells.