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

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

Doctors were replacing only one gene, and they only had to insert that gene into bone marrow cells.

The answer for 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?

Diabetes, since the target cells are found only in the pancreas

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