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Lee Sweeney, PhD

Myology Institute
University of Florida
1200 Newell Drive
ARB R5-234
Gainesville, Florida, United States

Dr. Lee Sweeney is presently the Director of Myology Institute, University of Florida. His research interests are focused on molecular motors of the myosin superfamily. Most notable among his accomplishments on molecular motors are the first visualization of structural rearrangement of the myosin lever arm, a detailed analysis of how processive myosins are engineered, a demonstration of the structural changes induced by actin-binding and nucleotide release, and the discovery and molecular dissection of the only known reverse-direction myosin.

Dr. Sweeney is actively developing therapeutics for rare diseases that include both small molecule and gene therapy approaches. He serves as a consultant to a number of industry therapeutic development efforts for Duchenne muscular dystrophy and spinal muscular atrophy. In 2007, he and his collaborators at PTC Therapeutics (a small NJ biotech company) published the development of a compound (PTC 124 or ataluren) that allows read-through of nonsense mutations (premature stop codons) in a variety of genetic disease models. The drug is in clinical trials for Duchenne muscular dystrophy and Cystic Fibrosis. On May 23, 2014, ataluren was granted conditional European approval for the treatment of Duchenne muscular dystrophy (DMD), making it the first approved drug for this disease.

Dr. Sweeney is well-known in the popular press for his gene-therapy approaches to permanently block the loss of age-related muscle size and strength in mice. The technique suggests that therapies for humans could reverse the feebleness associated with old age or slow the muscle-wasting effects of muscular dystrophies.


Representative Publications:

Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy

A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Supraphysiological levels of GDF11 induce striated muscle atrophy

Disease-modifying effects of orally bioavailable NF-κB inhibitors in dystrophin-deficient muscle