Its promise was illustrated in 1990 when a 4-year-old girl named Ashanti DeSilva became the world’s first successful gene-therapy patient. Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency. Her identity revealed after years m the . In September 1990, four-year-old Ashanti DeSilva presented with Severe combined immunodeficiency syndrome that made her vulnerable to even the mildest infections. It is a countenance hke that of ]~cob . There, over the course of twelve days, Anderson and his colleagues Michael Blaese and Kenneth Culver slowly extracted some of Ashanti's blood cells. The treatment did not cure the near-total immune dysfunction that Ashanti suffered, but nonetheless it pulled her out of danger, allowed her to enroll in school, and illustrated the merit of treating genetic diseases at their most basic level. protocol with therapeutic intent. At the age of 14, Ashanti landed her first recording contract, with Jive Records. trans~er . Ashanti and her parents flew to the NIH Clinical Center at Bethesda, Maryland. Ashanti was born with adenosine deaminase defi ciency (ADA). Without that enzyme, DeSilva was likely to die a premature death. fi~st \ subject enrolled in a human gene . proto~ol, she stands as a symbol of hope and promise. 1990 First approved gene therapy trial. Ashanti's immune system was partially restored by the therapy. As Raj DeSilva told the Houston Chronicle, "What choice did we have?" Given these odds, her parents opted to try the therapy. Ashanti and her parents flew to the NIH Clinical Center at Bethesda, Maryland. Trials and tribulations of gene therapy. Sontag, his worried face gracing . It is the face of Ashanti De Silva, her identity long obscured behind the veil of confidentiality, who at four years old was the . Dr French Anderson with 4 year old Ashanti DeSilva. Soon afterward, Ashanti's parents entered their daughter in a local talent show, which she handily won. As Raj DeSilva told the Houston Chronicle, "What choice did we have?" People with this genetic disorder do not produce an enzyme necessary for immune system function. From then on, Ashanti's mother has acted as her co–manager. In September 1990, four-year-old Ashanti de Silva became famous in the scientifi c and medical communities as the world’s fi rst gene therapy patient. This label also featured such top artists as the Backstreet Boys, 'N Sync, and Britney Spears. The defective gene of the patient's blood cells was replaced by the functional variant. A single genetic defect- like a typo in a novel- left DeSilva unable to produce an important enzyme.

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