Subretinal Gene Therapy of Mice With Bardet-Biedl Syndrome Type 1

PURPOSE. To study safety and efficacy of subretinal adeno-associated virus (AAV) vector AAV-Bbs1 injection for treatment of a mouse model of Bardet-Biedl syndrome type 1 (BBS1). METHODS. Constructs containing a wild-type (WT) Bbs1 gene with and without a FLAG tag in AAV2/5 vectors were generated. Vi...

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Hauptverfasser: Seo, Seongjin, Mullins, Robert F., Dumitrescu, Alina V., Bhattarai, Sajag, Gratie, Daniel, Wang, Kai, Stone, Edwin M., Sheffield, Val, Drack, Arlene V.
Format: Artigo
Sprache:Inglês
Veröffentlicht: The Association for Research in Vision and Ophthalmology 2013
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Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3771708/
https://ncbi.nlm.nih.gov/pubmed/23900607
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1167/iovs.13-11673
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