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Nonviral delivery techniques for in vivo prime editing

US National Heart Lung and Blood Institute grant open #nih-5R01HL162564-04

Summary

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

Nonviral delivery techniques for in vivo p…
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