It has been suggested that gene therapy is a possible treatment for muscular dystrophy - VCE - SSCE Biology - Question 16 - 2010 - Paper 1
Question 16
It has been suggested that gene therapy is a possible treatment for muscular dystrophy.
Expected benefits if this technique is successfully used include the
A. inabi... show full transcript
Worked Solution & Example Answer:It has been suggested that gene therapy is a possible treatment for muscular dystrophy - VCE - SSCE Biology - Question 16 - 2010 - Paper 1
Step 1
A. inability of the introduced allele being passed on to offspring.
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Answer
Gene therapy typically aims to address existing genetic disorders rather than preventing their inheritance. Therefore, this option is not a primary benefit of gene therapy.
Step 2
B. introduced DNA remaining functional for a short time in cells.
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Answer
While there are instances in which temporary expression of a gene can be beneficial for treatment, the ideal outcome of gene therapy is to provide a lasting solution. Thus, this option does not represent a significant benefit.
Step 3
C. integration of the introduced allele into many different gene loci.
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Successful gene therapy does not generally involve random integration of the introduced allele; instead, targeted integration is preferred to avoid disrupting essential genes.
Step 4
D. production of normal dystrophin protein in genetically affected individuals.
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This is the key benefit of gene therapy for muscular dystrophy, as the introduction of a correct copy of the dystrophin gene aims to enable the synthesis of normal dystrophin protein, thus alleviating the symptoms of the disease.
