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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 issue that scientists may be cautious about regarding germline gene therapy is that it is unethical. This approach could have long-term effects that are not yet known, particularly since it affects individuals who are not yet born, leaving them without a choice regarding their genetic makeup.
Step 2
What evidence, at a cellular level, would confirm that the processes above were successful?
Answer
At a cellular level, evidence confirming the success of the above processes can include the correct production of the target protein and the functionality of that protein, indicating that the normal gene has been effectively incorporated and is functioning as intended.
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 cells that have been successfully altered will be injected back into the patient, thus minimizing the risk of unintended effects that may occur with in-vivo methods, where the viral vector can potentially affect all cells regardless of whether they are altered.