Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases

Adeno-associated viral (AAV) vectors containing cone-specific promoters have rescued cone photoreceptor function in mouse and dog models of achromatopsia, but cone-specific promoters have not been optimized for use in primates. Using AAV vectors administered by subretinal injection, we evaluated a s...

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Bibliografiske detaljer
Udgivet i:Hum Gene Ther
Main Authors: Ye, Guo-Jie, Budzynski, Ewa, Sonnentag, Peter, Nork, T. Michael, Sheibani, Nader, Gurel, Zafer, Boye, Sanford L., Peterson, James J., Boye, Shannon E., Hauswirth, William W., Chulay, Jeffrey D.
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2016
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4741229/
https://ncbi.nlm.nih.gov/pubmed/26603570
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.130
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