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Genetic Linkage

Gene Therapy Update: Remembering Jesse Gelsinger

Like the mythological phoenix bird, gene therapy has risen from the ashes and is spreading its wings.

September 17 marked 20 years since the death of 19-year-old Jesse Gelsinger in a gene therapy trial. That tragedy halted the fledgling field, with the outlook worsening when, soon after, boys with an inherited immune deficiency developed leukemia when a gene therapy went off course. The momentum that had been slowly building since the first clinical trial in 1990 fizzled.

 

A Slow Comeback

 

Researchers rebuilt the viruses that ferry in working copies of genes, and gradually clinical trials resumed. But it took until late 2017 for the first FDA approval of a gene therapy: Luxturna, for blindness due to mutation of a gene called RPE65.

 

My book The Forever Fix: Gene Therapy and the Boy Who Saved It, from 2012, chronicles the history of the field as a backdrop to the Luxturna story. The "boy," Corey Haas, was 8 when he was treated in 2008. He's made amazing progress.

 

To continue reading go to my DNA Science blog at Public Library of Science. 

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