AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6β-deficient Dogs

We previously reported that subretinal injection of AAV2/5 RK.cpde6β allowed long-term preservation of photoreceptor function and vision in the rod-cone dysplasia type 1 (rcd1) dog, a large animal model of naturally occurring PDE6β deficiency. The present study builds on these earlier findings to pr...

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Foilsithe in:Mol Ther
Main Authors: Pichard, Virginie, Provost, Nathalie, Mendes-Madeira, Alexandra, Libeau, Lyse, Hulin, Philippe, Tshilenge, Kizito-Tshitoko, Biget, Marine, Ameline, Baptiste, Deschamps, Jack-Yves, Weber, Michel, Le Meur, Guylène, Colle, Marie-Anne, Moullier, Philippe, Rolling, Fabienne
Formáid: Artigo
Teanga:Inglês
Foilsithe: Nature Publishing Group 2016
Ábhair:
Original Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4881772/
https://ncbi.nlm.nih.gov/pubmed/26857842
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.37
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