Dr. Anh H. Pham from University of Miami School of Medicine Awarded $90,000 Knights Templar Eye Foundation Grant for CRISPR Based Mitochondrial Optic Neuropathy Treatment Research

Dr. Anh H. Pham from the Bascom Palmer Eye Institute, University of Miami School of Medicine, Miami, Florida was awarded a $90,000 grant entitled: Development of a Mitochondrial CRISPR Therapy for the Treatment of Mitochondrial Optic Neuropathy.

Mitochondria are unique organelles that have genetic content and serve as the powerhouse of the cell. Mutations in mitochondrial DNA (mtDNA) have been associated with many neuromuscular disorders and irreversible blindness in children for which there is no effective treatment or cure. Dr. Pham is adapting the novel gene editing technology known as CRISPR towards eliminating mutations in mtDNA. She is exploring two modalities for delivering the necessary components of CRISPR into the mitochondria. Dr. Pham is investigating the efficacy of CRISPR for treating several cellular models of pediatric ophthalmic diseases, including Leber Hereditary Optic Neuropathy (LHON), Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS), and Leigh syndrome with ophthalmoplegia. Successful demonstration of CRISPR for targeting mitochondrial DNA will open a new therapeutic avenue for these rare and incurable diseases.