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Breakthrough

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.

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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Muscular Dystrophy – Trial Could Lead to New Treatment

February 25, 2015

University of Florida Health researchers are participating in a key late-stage clinical trial that could lead to a new therapy for some children with Duchenne.

Keira Grace Foundation – Share the Cure 2015

February 13, 2015

Share the Cure 2015 Bring new life to children with cancer for 10 years! Keira Grace Foundation invites you to Cocktails, Dinner & Dancing Saturday, February 28, 2015 7:00 – 11:00pm...

Stop Childrens Cancer Bear-A-Thon

February 10, 2015

Help pediatric patients battle cancer ONE BEAR AT A TIME! Wednesday, February 11, 2015 is the 16th K COUNTRY/Stop Children’s Cancer Bear-A-Thon. Your generous donation of $40 to Stop Children’s...

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