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

1.Rhys Evans suffered from X chromosome linked severe combined immunodeficiency disorder, popularly known as “baby in the bubble” syndrome.The standard treatment for the condition is a bone marrow transplant but he had no donors.His parents then opted for gene therapy.The main reason for success in this story according to me is that Doctors were replacing only one gene, and they only had to insert that gene into bone marrow cells.

2. In people with cystic fibrosis , a defective gene causes a thick, buildup of mucus in the lungs, pancreas, and other organs.Because this is the disorder of a single gene that would be the easiest to treat through gene therapy.

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