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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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December Department Highlights

December 16, 2014

UF Health Child Life Program Helps Bring Christmas Cheer to Patients The UF Health Child Life Program was established to help children and families cope with the stress and anxiety […]

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