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...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7887332/
https://www.ncbi.nlm.nih.gov/pubmed/33665223
http://dx.doi.org/10.1016/j.omtm.2020.12.014

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7887332/
https://www.ncbi.nlm.nih.gov/pubmed/33665223
http://dx.doi.org/10.1016/j.omtm.2020.12.014

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