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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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Detalles Bibliográficos
Autores principales: Beltran, William A., Boye, Sanford L., Boye, Shannon E., Chiodo, Vince A., Lewin, Alfred S., Hauswirth, William W., Aguirre, Gustavo D.
Formato: Texto
Lenguaje:English
Publicado: 2010
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2914811/
https://www.ncbi.nlm.nih.gov/pubmed/20428215
http://dx.doi.org/10.1038/gt.2010.56