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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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Autors principals: Beltran, William A., Boye, Sanford L., Boye, Shannon E., Chiodo, Vince A., Lewin, Alfred S., Hauswirth, William W., Aguirre, Gustavo D.
Format: Artigo
Idioma:Inglês
Publicat: 2010
Matèries:
Accés en línia: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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