@article{46164c092e104856811f9d84c52cba7d,
title = "Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice",
abstract = "Recently, the benefits of AAV9 gene therapy were reported in a mouse model of CLN6-Batten disease, a rare neuropediatric disease. Here, the authors show that intracerebroventricularly delivered AAV9 prevents disease pathology in the visual centers of the brain, expresses in the retina, and partially preserves vision in Cln6nclf mice.",
keywords = "AAV9, Batten disease, CLN6, Gene therapy, ICV, NCL, retina, vision",
author = "White, {Katherine A.} and Nelvagal, {Hemanth R.} and Poole, {Timothy A.} and Bin Lu and Johnson, {Tyler B.} and Samantha Davis and Pratt, {Melissa A.} and Jon Brudvig and Assis, {Ana B.} and Shibi Likhite and Kathrin Meyer and Kaspar, {Brian K.} and Cooper, {Jonathan D.} and Shaomei Wang and Weimer, {Jill M.}",
note = "Publisher Copyright: {\textcopyright} 2021 The Authors",
year = "2021",
month = mar,
day = "12",
doi = "10.1016/j.omtm.2020.12.014",
language = "English",
volume = "20",
pages = "497--507",
journal = "Molecular Therapy - Methods and Clinical Development",
issn = "2329-0501",
}