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Genetic Linkage

The Past and the Future of Gene Therapy

Corey Haas and Hannah Sames sign their photographs in "The Forever Fix: Gene Therapy and the Boy Who Saved It."
Yesterday I had two very special guests at my book talk and signing at the Schenectady library: Eleven-year-old Corey Haas, who is “the boy who saved gene therapy” in the metaphorical phrase in the book title, and eight-year-old Hannah Sames, who will have gene therapy.

Corey and Hannah represent gene therapy’s immediate past and future. They put faces on a once-moribund biotechnology reborn after a series of tragic errors and failures. They are also remarkable children: bright, poised, aware, and charming. They are making history.

Just four days after his gene therapy to treat Leber congenital amaurosis, a form of inherited blindness, Corey became able to see – very dramatically, at the Philadelphia zoo. It was September 2008, nine years after an 18-year-old, Jesse Gelsinger, died in a gene therapy experiment in the same city.

Hannah is in the “natural history” testing phase for her gene therapy for giant axonal neuropathy (GAN), which will happen sometime in the next year or two, thanks to her parents’ relentless fundraising and a team of scientist-physicians as amazing as Corey’s. The gene therapy trial for GAN will pave the way for patients with other conditions affecting the motor neurons, with gene delivery into the spinal cord.

Among Corey and Hannah’s well-wishers is Paul Gelsinger, father of Jesse, who unintentionally sacrificed his life “to help the babies” who die of his metabolic condition.

“I am thankful that there is a great deal more honesty and oversight in the field now as a result of what happened to Jesse… I guess that’s his real legacy. Feel free to tell anyone that I am glad there have been some breakthroughs and that I hope many more follow,” he e-mailed me just a few days ago, after reading my book.

Corey and Hannah, I am so proud to know you! And I wish I had known you, Jesse.

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