Annual Funded Grants – CRISPR and Prime Editing Technologies
Research News
Funded grants for CRISPR and prime editing approaches.
The Foundation Fighting Blindness is proud to announce funding for several projects based on CRISPR and prime editing technologies.
Stephen Tsang – $200,000
Columbia University
“ARMS2/HTRA1 in non–cell-autonomous oxidative and anti-inflammatory therapeutic targeting”
Drs. Tsang and Olah will use CRISPR to identify the causative allele of AMD pathologies and investigate the stress signals of microglia (resident immune cells of the eye,) in AMD that might be treatable as part of a therapeutic strategy to reduce AMD-related cell death. They will also explore whether the presence of at least one low-risk ARMS2/HTRA1 allele maintains oxidative, anti-inflammatory, and overall cellular health in microglia.
Qin Liu – $100,000
Mass Eye and Ear
“Development of precise correction of c.2299delG mutation in the USH2A gene.”
Dr. Liu and her team will investigate the potential for using prime editing to correct the c.2299delG mutation in the USH2A. Traditional gene therapy is difficult due to the large size of the USH2A gene and the cargo capacity of delivery systems (viruses). Prime editing offers a precise method to correct single gene mutations. This research effort will focus on the feasibility of delivering prime editing components via an adeno-associated virus (AAV) to repair this mutation in a humanized mouse model of USH2A disease.
Peter M.J. Quinn – $100,000
University of Pennsylvania
“Prime editing for Peripherin-2 (PRPH2) inherited retinal dystrophies”
Dr. Quinn is testing a prime editing technique for multiple mutations in PRPH2 using patient-derived retinal organoids. Prime editing is a gene editing technique that splices directly at the site of the mutations and switches out a mutated copy of the gene with a healthy copy. Successful completion of this project will establish a preclinical pathway for proof-of-concept for PRPH2 prime editing therapeutics and lay the foundation for the same strategy to be applied to other IRDs.
Krzysztof Palczewski – $159,868
The Regents of the University of California, Irvine
“Precision genome editing in humanized mice expressing mutant peripherin-2”
Dr. Palczeski aims to create a mouse carrying the human PRPH2 gene in place of the mouse gene, which will allow researchers to test genome editing therapies for PRPH2-associated retinal diseases.
Krzysztof Palczewski – $300,000
The Regents of the University of California, Irvine
“Correcting previously untreatable retinal degenerative diseases using twin prime editing”
Dr. Palczewski seeks to advance a novel gene editing technology called twin prime editing for treating a model of Stargardt disease and develop a twin prime editing framework to address other inherited retinal degenerative diseases.
