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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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Bibliografiska uppgifter
Huvudupphovsmän: Beltran, William A., Boye, Sanford L., Boye, Shannon E., Chiodo, Vince A., Lewin, Alfred S., Hauswirth, William W., Aguirre, Gustavo D.
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2010
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Länkar: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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