the first clinical gene therapy widely accepted as successful was done for the treatment of beta-thalassemia.
the first clinical gene therapy widely accepted as successful was done for the treatment of beta-thalassemia.
first clinical gene therapy widely accepted as successful was done for the treatment of?
beta-thalassemia
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. 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
after extensive research on animals throughout the 1980s and a 1989 bacterial gene tagging trial on humans, the first gene therapy widely accepted as a success was demonstrated in a trial that started on 14 september 1990, when ashi desilva was treated for ada-scid.









































the first somatic treatment that produced a permanent genetic change was initiated in 1993. the goal was to cure malignant brain tumors by using recombinant dna to transfer a gene making the tumor cells sensitive to a drug that in turn would cause the tumor cells to die.
gene therapy