Advancing precision ace-tRNA readthrough technology for the treatment of choroideremia
JOHN OSTER LEADERSHIP AWARD
David Gamm, MD, PhD
Professor, Ophthalmology and Visual Sciences Director
McPherson Eye Research Institute University of Wisconsin, Madison, WI
Christopher Ahern, PhD
Professor, Molecular Physiology and Biophysics
University of Iowa, Carver College of Medicine, Iowa City, IA
Lay Abstract
Engineered transfer RNA (ace-tRNA) technology is a novel and highly effective strategy to overcome the wide array of nonsense mutations affecting one-third CHM patients. The proposal will determine the efficacy, accuracy, and stability of next-generation ace-tRNA molecules to restore substantial production of REP1 in human induced pluripotent stem cell (hiPSC)-derived retinal pigmented epithelial cells and retinal organoids. These pre-clinical studies are designed to support a future investigational new drug application (IND) that will employ ace-tRNA technology for the treatment of CHM.
Key Findings
This research is currently underway. Key findings will be published upon research conclusion.
CRF is registered as a US 501(c)3 nonprofit organization EIN: 04-3511754
Our mission is to raise funds in support of scientific research leading to a treatment or cure of choroideremia, a hereditary retinal-degenerative disease that causes blindness; to educate people affected by the disease; and to inform the public.
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