NIH R01 · 2024
Nonviral delivery techniques for in vivo prime editing
Gene editing is a promising strategy for treating or even permanently curing genetic diseases. In particular, a new technique called prime editing has the potential to make small targeted insertions, deletions, and substitutions with very high potential coverage of known disease- causing mutations, and while minimizing dangerous double-stranded breaks in DNA. In order to realize this potential, robust delivery strategies must be developed to deliver prime editing tools efficiently to disease-relevant organs. One such delivery strategy is lipid nanoparticle delivery of RNA and/or protein-based prime editing components. LNPs are nonviral, nontoxic, and clinically validated delivery tools.…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.