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Question
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? gauchers disease, where fat deposits accumulate in cells throughout the body cystic fibrosis, since the target cells are found in both the lungs and the gi tract diabetes, since the target cells are found only in the pancreas down syndrome, since there is an extra copy of chromosome 21 in every cell of the body
Gene therapy is more straightforward when the target cells are in a single - organ. Diabetes has target cells only in the pancreas, while Gaucher's disease affects cells throughout the body, cystic fibrosis affects cells in multiple major organs (lungs and GI tract), and Down syndrome affects every cell in the body due to an extra chromosome. Delivering the corrected gene to a single - organ's cells is relatively easier than to cells in multiple organs or throughout the body.
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diabetes, since the target cells are found only in the pancreas