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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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National Breastfeeding Awareness Month

August 29, 2014

Having a baby is a joyous moment for many parents. But something very important to decide is whether to breastfeed or bottlefeed. Dr. Sandra Sullivan sits down to discuss the topic.

Gene therapist on patient unity

August 29, 2014

Patients are fighting back against the lack of attention that’s given to their diseases, a leading expert in genetic and rare diseases says.

August Department Highlights

August 26, 2014

Dairy Queen helps make miracles for Children’s Miracle Network Hospitals and UF Health Shands Children’s Hospital Through the generosity of two Dairy Queen franchise operators from South Florida,...

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