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.
|Number of pages||11|
|Journal||Molecular Therapy - Methods and Clinical Development|
|State||Published - Mar 12 2021|
- Batten disease
- Gene therapy