Gene Therapy Review: Gene and Cell Therapy Resource
How “The Forever Fix: Gene Therapy and the Boy Who Saved It” Wrote Itself
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- Category: Gene Therapy Books
- Published on Thursday, 17 May 2012 06:44
- Written by Ricki Lewis
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When I met smiling, 9-year-old Corey Haas on a dazzling Saturday morning in early December, 2009, I knew that the time had finally arrived to write a book about gene therapy for the general public. St. Martin’s Press published “The Forever Fix: Gene Therapy and the Boy Who Saved It,” in March, 2012. It was a long time coming – as was the field of gene therapy itself.
I’d been writing about gene therapy since the beginning. In September 1990, at the NIH, 4-year-old Ashi DeSilva received her own altered white blood cells to treat ADA deficiency. Two other Septembers form the high and low points of my book: Corey’s amazing ability to see at the Philadelphia zoo, four days after gene therapy for Leber congenital amaurosis type 2 in 2008, and 18-year-old Jesse Gelsinger’s death, four days after his gene therapy to treat ornithine transcarbamylase deficiency, in the same city in 1999.
Clinical Data Published for a Phase I AADC-deficiency Trial Using a Gene Therapy Manufactured by Florida Biologix
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- Category: Press Releases
- Published on Thursday, 17 May 2012 06:35
- Written by Chris Aytug
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ALACHUA, FL May 16, 2012 - Florida Biologix®, a full-service biopharmaceutical development and contract manufacturing organization, provided cGMP manufacturing services to National Taiwan University Hospital under a contract signed in March 2008. Florida Biologix produced, purified, and tested a clinical batch of their novel gene therapy for a Phase I clinical trial of a rare genetic disease – AADC deficiency. The trial was led by Dr. Paul Wuh-Liang Hwu. Results of the clinical trial were published in the May 16th issue of Science Translational Medicine.
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What is Gene Therapy?
Gene Therapy is the transfer of genetic material (gene transfer) to dysfunctional cells to correct a deficiency in the DNA or genome of a patient. Cell Therapy, such as stem cell therapy, can also be used to achieve these goals. These approaches can be applied to genetic disorders as well as diseases acquired over the lifetime of an individual, such as cancer or infection, to confer a specific property to the cell allowing it to combat the disease.