Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice

Batten disease is a family of rare, fatal, neuropediatric diseases presenting with memory/learning decline, blindness, and loss of motor function. Recently, we reported the use of an AAV9-mediated gene therapy that prevents disease progression in a mouse model of CLN6-Batten disease (Cln6(nclf)), re...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther Methods Clin Dev
Prif Awduron: White, Katherine A., Nelvagal, Hemanth R., Poole, Timothy A., Lu, Bin, Johnson, Tyler B., Davis, Samantha, Pratt, Melissa A., Brudvig, Jon, Assis, Ana B., Likhite, Shibi, Meyer, Kathrin, Kaspar, Brian K., Cooper, Jonathan D., Wang, Shaomei, Weimer, Jill M.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Gene & Cell Therapy 2021
Pynciau:
Original Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC7887332/
https://ncbi.nlm.nih.gov/pubmed/33665223
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.12.014
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