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Josh’s Fight to Cure FA

Please watch this video of Josh and donate whatever you can to help him and the thousands of people worldwide with FA (Friedreich’s ataxia), a debilitating, life-shortening, degenerative neuro-muscular disorder. We […]



Using gene transfer techniques pioneered by University of Florida faculty, Taiwanese doctors have restored some movement in four children bedridden with a rare, life-threatening neurological disease.

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A Pioneer in Gene Therapy

The Powell Gene Therapy Center has been a pioneer in several ways:

  • Discovery of the unique life cycle of AAV (Dr. Kenneth Berns)
  • First use of AAV to deliver genes to cells in culture (Dr. Nicholas Muzyczka)
  • First use of AAV in animals and humans (Dr. Terry Flotte)
  • First use of AAV to in models of muscle and heart disease (Dr. Barry Byrne)
  • First use of AAV to reverse blindness in dogs with a genetic disease (Dr. Bill Hauswirth)
  • Active clinical trials in CF patients since 1996, including two that are currently enrolling patients (Dr. Terry Flotte)
  • A clinical trial in AAT deficiency (genetic emphysema) to begin in early 2004 (Drs. Terry Flotte and Mark Brantly)
  • The PGTC has competed successfully for federal funding in numerous arenas

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Give Kids the World Silent Auction

September 24, 2015

Thursday, September 24, 2015 @5:00 PM Hilton University of Florida Conference Center Give Kids the World Village (GKTW) is a non-profit organization for children with terminal and life-threatening...