Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice
Sign Up to like & getrecommendations! Published in 2020 at "Nature Communications"
DOI: 10.1038/s41467-019-14181-3
Abstract: Supplementing wildtype copies of functionally defective genes with adeno-associated virus (AAV) is a strategy being explored clinically for various retinal dystrophies. However, the low cargo limit of this vector allows its use in only a… read more here.
Keywords: replacement; aav; wildtype; number photoreceptors ... See more keywords