Which of the following do you think contributed the most to the success of Rhys’

Question

Which of the following do you think contributed the most to the success of Rhys’ gene therapy?

Theoretically, which of the following diseases should be the easiest to treat through gene therapy, considering the targeted organs and tissues where the corrected gene must be delivered?

Rhys’ illness was never severe or life-threatening. Rhys was very young at the time of the gene therapy procedure. Doctors were able to supplement his gene therapy with numerous rounds of bone marrow transplants. Doctors were replacing only one gene, and they only had to insert that gene into bone marrow cells.

Explanation / Answer

In rhys case he has severe immune disorder, he does have immune system of it so called baby in bubble. This disorder is x linked. This was the first successful gene therapy in u. K. And in world second.

In this therapy dr. Did gene treatment with a virus and manuplate it and reinserted the normal gene. So its most likely reason for success is doctors were replacing only one gene and they only had to insertthat gene into bone marow cell.

Ans of second ques is.. Gauchers disease, where fat deposits accumulate in cells throughout body. After treatibg it with virus this will break fat into bloid leading to no fat deposition.

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