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Ricki Lewis

Genetic Linkage

Second Gene Therapy Nears Approval in Europe: Lessons for CRISPR?

April 20, 2016
Tags: gene therapy, ADA deficiency, bubble boy
CRISPR-Cas9 gene editing has been around not even 4 years, and people are avidly discussing its promises and perils. That’s great. But consider the historical backdrop.

April 1, the European Medicine Agency’s (EMA) Committee for Medicinal Products recommended for marketing approval a second gene therapy. “Strimvelis” treats adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID) and was developed at the San Raffaele Telethon Institute for Gene Therapy in Milan and GlaxoSmithKline. Regulatory approval is expected within a few months.

I wonder how many people realize, especially those fearful of how gene editing might be misused, that the gene therapy that is nearing approval actually entered clinical trials 26 years ago? Read More 
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SCID-X1 Gene Therapy, Take 2

October 12, 2014
Tags: gene therapy, bubble boy, SCID-X1, ADA deficiency, forever fix
David Vetter had SCID-X1 and became known as the bubble boy. (NASA)
Beneath all the bad news about viruses this week lies a good virus: the one that underlies gene therapy for X-linked severe combined immunodeficiency (SCID-X1),

Altered viruses are the vehicles that transfer healthy human genes into the cells of people in whom the genes aren’t working, providing a slew of new “forever fixes.” Nearly 100 gene transfer protocols are now in late-stage clinical trials. Read More 
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Gene Therapy and the 10,000-Hour Rule

January 4, 2012
Tags: gene therapy, The Forever Fix: Gene Therapy and the Boy Who Saved It, Malcolm Gladwell, 10, 000-hour rule, Ashi DeSilva, Biology Digest, William French Anderson, Donald Kohn, ADA deficiency, Leber congenital amaurosis, Kalydeco, ivacaftor, Vertex Pharmaceuticals, cystic fibrosis, severe combined immune deficiency, Ricki Lewis, Corey Haas, Jesse Gelsinger
“Breakthroughs” in biomedicine are rarely that – they typically rest on a decade or more of experiments. Consider gene therapy.

I just unearthed an article from the December 1990 issue of Biology Digest, "Gene Therapy." I wrote it a mere two months after the very first gene therapy experiment, the much-publicized case of 4-year-old Ashi DeSilva,  Read More 
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