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Gene therapy can be used to introduce normal genes into cells to replace missing or defective genes in order to treat or prevent disease - VCE - SSCE Biology - Question 11 - 2016 - Paper 1
Question 11
Gene therapy can be used to introduce normal genes into cells to replace missing or defective genes in order to treat or prevent disease. Scientists have taken a num... show full transcript
Worked Solution & Example Answer:Gene therapy can be used to introduce normal genes into cells to replace missing or defective genes in order to treat or prevent disease - VCE - SSCE Biology - Question 11 - 2016 - Paper 1
Step 1
Identify one issue that would make scientists cautious about using germline gene therapy.
Answer
One significant concern regarding germline gene therapy is that it is often viewed as unethical. This is largely due to the implications of altering genes in embryos, which could affect future generations without their consent. Additionally, there is a risk the gene could inadvertently be inserted in an incorrect location within the genome, leading to unintended consequences such as negative impacts on other genes.
Step 2
What evidence, at a cellular level, would confirm that the processes above were successful?
Answer
At a cellular level, the confirmation of successful gene therapy processes would involve demonstrating that the correct protein is being produced by the altered cells. This could be assessed by analyzing the expression levels of the gene and verifying through biochemical assays that the protein is functional and performing its intended role in the cell.
Step 3
What is one advantage that ex-vivo gene therapy has over in-vivo gene therapy?
Answer
One advantage of ex-vivo gene therapy is that only the cells that have been successfully altered using the viral vector are injected back into the patient. This reduces the risk of the viral vector affecting non-target cells, providing a more controlled and potentially safer approach compared to in-vivo methods, where the vectors are directly injected into the patient.