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28 wrz 2021 · After another 18 years of research, the NIH undertook the first approved gene therapy clinical trial. The trial involved Ashanti DeSilva (Figure 1), a four-year-old child with a severe combined immunodeficiency caused by a mutation in the gene encoding for the adenosine deaminase enzyme.
14 wrz 2018 · Ashanti DeSilva, born in 1986, was 26-months-old when she was diagnosed with SCID after being afflicted by an almost ceaseless series of infections. She suffered from adenosine deaminase (ADA) deficiency, a rarer and slightly less severe form of SCID compared to David Vetter's X-linked SCID variant. W.
14 lis 2018 · In recent decades, clinical gene therapy trials have underpinned the research that has significantly contributed to advancements in the development of therapies for this and other diseases – and these trials can be traced back to a young girl named Ashanthi DeSilva.
1 maj 2021 · Ashanthi de Silva was the first human to be treated successfully with gene therapy. At the time, in 1990, she was a 4-year-old living with severe combined immunodeficiency (SCID), which is caused by insufficient levels of the enzyme adenosine deaminase (ADA).
14 lis 2018 · In recent decades, clinical gene therapy trials have underpinned the research that has significantly contributed to advancements in the development of therapies for this and other diseases – and these trials can be traced back to a young girl named Ashanthi DeSilva.
1 sty 2020 · Four-year-old Ashanti De Silva received multiple infusions of her own peripheral blood T lymphocytes that had been transduced with a human ADA gene-containing RV vector. Engraftment was successful, and immunological improvement occurred but did not allow discontinuation of enzyme replacement therapy (ERT) ( Blaese et al., 1995 ).
10 mar 2016 · Ashanti DeSilva, the first patient for whom treatment with gene therapy was authorized by the FDA and NIH’s RAC, has few memories of her gene therapy in 1990, but many of R. Michael...
