CRISPR-Based Gene editing for Choroideremia

The outcomes of viral-mediated gene therapy trials in Ophthalmology, including Choroideremia, have uncovered some unforeseen problems, such as the most recent Phase III clinical trial sponsored by Biogen, which failed to meet the endpoints. Traditional gene therapy typically involves viral-mediated gene supplementation rather than gene correction, but this strategy has revealed several limitations. In contrast, the novel cutting-edge techniques of genome editing have the potential to directly correct one’s genetic code, addressing several limitations faced by traditional viral-based gene therapy. Prime editing (PE) is a gene-editing technology that mediates targeted insertions, deletions, and all 12 possible base-to-base conversions. We use PE to correct CHM mutations using mRNA and non-viral particles to deliver the PE components to the retina.