Krabbe disease successfully treated via monotherapy of intrathecal gene therapy

Authors

Allison M Bradbury, University of Pennsylvania
Xuntian Jiang, Washington University School of Medicine in St. Louis
Mark S Sands, Washington University School of Medicine in St. Louis
Daniel S Ory, Washington University School of Medicine in St. Louis
et al

Journal

The Journal of Clinical Investigation

Publication Date

2020

Volume

130

Issue

9

First Page

4906

Last Page

4920

Document Type

Open Access Publication

DOI

10.1172/JCI133953

Rights and Permissions

Bradbury AM, Bagel JH, Nguyen D, Lykken EA, Pesayco Salvador J, Jiang X, Swain GP, Assenmacher CA, Hendricks IJ, Miyadera K, Hess RS, Ostrager A, ODonnell P, Sands MS, Ory DS, Shelton GD, Bongarzone ER, Gray SJ, Vite CH. Krabbe disease successfully treated via monotherapy of intrathecal gene therapy. J Clin Invest. 2020 Sep 1;130(9):4906-4920. doi: 10.1172/JCI133953.

Recommended Citation

Bradbury, Allison M; Jiang, Xuntian; Sands, Mark S; Ory, Daniel S; and et al, "Krabbe disease successfully treated via monotherapy of intrathecal gene therapy." The Journal of Clinical Investigation. 130, 9. 4906 - 4920. (2020).
https://digitalcommons.wustl.edu/open_access_pubs/9518