Lebers Hereditary Optic Neuropathy (LHON), is a subtype of - TopicsExpress



          

Lebers Hereditary Optic Neuropathy (LHON), is a subtype of mitochondrial disease due to mtDNA mutations that result in rapid, permanent and bilateral loss of vision primarily in males between the ages 20-40. Dr. John Guy at the Bascom Palmer Eye Institute at the University of Miami has received NIH funding to develop gene therapy to replace the defective gene in retinal cells. His new drug which which targets the retina and carries a healthy gene via an adenoviral vector, AAV-ND4 gene IND#15941, was just approved by the FDA and he is beginning a phase I trial in April 2014 concluding in five years. If you or a loved one have LHON and are interested in the trial please contact Marla Bercuson at 305-326-6190 for more information.
Posted on: Mon, 14 Apr 2014 15:12:12 +0000

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