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rAAV2/5 gene-targeting to rods: dose-dependent efficiency and complications associated with different promoters

A prerequisite for using corrective gene therapy to treat humans with inherited retinal degenerative diseases that affect primarily rods is to develop viral vectors that target specifically this population of photoreceptors. The delivery of a viral vector with photoreceptor tropism coupled with a ro...

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Detalhes bibliográficos
Main Authors: Beltran, William A., Boye, Sanford L., Boye, Shannon E., Chiodo, Vince A., Lewin, Alfred S., Hauswirth, William W., Aguirre, Gustavo D.
Formato: Artigo
Idioma:Inglês
Publicado em: 2010
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2914811/
https://ncbi.nlm.nih.gov/pubmed/20428215
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2010.56
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