GENETICS MAJORS ONLY PLEAASE!
A gene therapy for ADA deficiency using stem cells is being tested. Gene therapy for muscular dystrophy uses immature muscle cells. What is the reasoning behind choosing these types of cells for the therapies? Are these therapies likely in vivo or ex vivo? Explain. (6 marks)
GENETICS MAJORS ONLY PLEAASE!
A gene therapy for ADA deficiency using stem cells is being tested. Gene therapy for muscular dystrophy uses immature muscle cells. What is the reasoning behind choosing these types of cells for the therapies? Are these therapies likely in vivo or ex vivo? Explain. (6 marks)
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Muscular dystrophy is an umbrella term for several genetic diseases that all cause increasing weakness of a person's muscles. Duchenne muscular dystrophy and Becker muscular dystrophy are different types of muscular dystrophy. Each one is caused by a different type of mutation in the gene coding for a protein that helps to prevent muscle cells from dying prematurely.
Duchenne muscular dystrophy is more severe. A child with Duchenne muscular dystrophy begins to show symptoms of loss of muscle function by about age 3 and needs to use a wheelchair by about age 10. A person with Duchenne muscular dystrophy typically dies as a young adult, due to failure of the muscles in the internal organs.
Becker muscular dystrophy is milder. Symptoms do not begin until age 12 or later, and the person can live into their 40s or 50s.
The table shows the main kinds of mutations that are responsible for these two types of muscular dystrophy. Complete the second column to indicate which type of muscular dystrophy you think would be caused by each type of mutation.
Type of Mutation | Type of Muscular Dystrophy |
Deletion Mutation -> # nucleotides deleted from mRNA is a multiple of 3 | |
Deletion Mutation -. # nucleotides deleted from mRNA is not a multiple of 3 | |
Point Mutation Ć stop codon |
Explain your reasoning.