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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Foilsithe in: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.
Formáid: Artigo
Teanga:Inglês
Foilsithe: Mary Ann Liebert, Inc. 2016
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Research Articles
Rochtain Ar Líne: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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