Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus.
Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus.
J. Flannery,S. Zolotukhin,3 Authors,W. Hauswirth
1997 · DOI: 10.1073/PNAS.94.13.6916
Proceedings of the National Academy of Sciences of the United States of America · 402 Citations
TLDR
High purified, helper virus-free rAAV vectors can achieve high-frequency tissue-specific transduction of terminally differentiated, postmitotic photoreceptor cells and suggest the feasibility of genetic therapy for retinal disease.