First clinical gene therapy was done for the treatment of?
The first attempt, an unsuccessful one, at gene therapy (as well as the first case of medical transfer of foreign genes into humans not counting organ transplantation) was performed by Martin Cline on 10 July 1980. Cline claimed that one of the genes in his patients was active six months later, though he never published this data or had it verified and even if he is correct, it's unlikely it produced any significant beneficial effects treating beta-thalassemia.
The first clinical gene therapy that was ultimately unsuccessful was done for the treatment of?
beta-thalassemia
Gene therapy
No context provided
First clinical gene therapy widely accepted as successful was done for the treatment of?
severe immune system deficiency ADA-SCID
Gene therapy
https://en.wikipedia.org/wiki/Gene%20therapy
The first approved gene therapy clinical research in the US took place on 14 September 1990, at the National Institutes of Health (NIH), under the direction of William French Anderson.[115] Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency. The defective gene of the patient's blood cells was replaced by the functional variant. Ashanti's immune system was partially restored by the therapy. Production of the missing enzyme was temporarily stimulated, but the new cells with functional genes were not generated. She led a normal life only with the regular injections performed every two months. The effects were successful, but temporary
Gene therapy
The first attempt, albeit an unsuccessful one, at gene therapy was performed by Martin Cline on July 10, 1980 in an attempt to treat beta-thalassemia. Cline claimed that one of the genes in his patients was active six months later, though he never published this data or had it verified. Additionally, even if he is correct, it's unlikely it produced any significant beneficial effects treating beta-thalassemia. Then, in 1990, the first approved gene therapy clinical research in the United States took place in September at the National Institutes of Health under the direction of William French Anderson. Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency. The defective gene of the patient's blood cells was replaced by the functional variant. Ashanti's immune system was partially restored by the therapy. Production of the missing enzyme was temporarily stimulated; however, the new cells with functional genes were not generated. She then led a normal life with only the regular injections performed every two months. The effects were successful though temporary.