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

Katherine A. White, Hemanth R. Nelvagal, Timothy A. Poole, Bin Lu, Tyler B. Johnson, Samantha Davis, Melissa A. Pratt, Jon Brudvig, Ana B. Assis, Shibi Likhite, Kathrin Meyer, Brian K. Kaspar, Jonathan D. Cooper, Shaomei Wang, Jill M. Weimer

Research output: Contribution to journalArticlepeer-review

8 Scopus citations

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.

Original languageEnglish
Pages (from-to)497-507
Number of pages11
JournalMolecular Therapy - Methods and Clinical Development
Volume20
DOIs
StatePublished - Mar 12 2021

Keywords

  • AAV9
  • Batten disease
  • CLN6
  • Gene therapy
  • ICV
  • NCL
  • retina
  • vision

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