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

Authors

Katherine A. White, Sanford Research, Sioux Falls
Hemanth R. Nelvagal, Washington University School of Medicine in St. Louis
Timothy A. Poole, Washington University School of Medicine in St. Louis
Bin Lu, Cedars-Sinai Medical Center, Los Angeles
Tyler B. Johnson, Sanford Research, Sioux Falls
Samantha Davis, Sanford Research, Sioux Falls
Melissa A. Pratt, Sanford Research, Sioux Falls
Jon Brudvig, Sanford Research, Sioux Falls
Ana B. Assis, University of California, Los Angeles
Shibi Likhite, Nationwide Children's Hospital
Kathrin Meyer, The Ohio State University
Brian K. Kaspar, The Ohio State University
Jonathan D. Cooper, Washington University School of Medicine in St. Louis
Shaomei Wang, Cedars-Sinai Medical Center, Los Angeles
Jill M. Weimer, Sanford Research, Sioux Falls

Journal

Molecular Therapy-Methods & Clinical Development

Publication Date

2021

Volume

20

First Page

497

Last Page

507

Document Type

Open Access Publication

DOI

10.1016/j.omtm.2020.12.014

Rights and Permissions

White, K. A., Nelvagal, H. R., Poole, T. A., Lu, B., Johnson, T. B., Davis, S., . . . Weimer, J. M. (2021). Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-batten disease mice. Molecular Therapy - Methods and Clinical Development, 20, 497-507. doi:10.1016/j.omtm.2020.12.014 This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

Recommended Citation

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; and Weimer, Jill M., "Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice." Molecular Therapy-Methods & Clinical Development. 20, 497 - 507. (2021).
https://digitalcommons.wustl.edu/open_access_pubs/10109