Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease

Authors

Gregory J Heller, University of Illinois at Chicago
Michael S Marshall, University of Illinois at Chicago
Yazan Issa, University of Illinois at Chicago
Jeffrey N Marshall, University of Illinois at Chicago
Duc Nguyen, University of Illinois at Chicago
Emily Rue, Oregon State University
Koralege C Pathmasiri, University of Illinois at Chicago
Miriam S Domowicz, University of Chicago
Richard B van Breemen, Oregon State University
Leon M Tai, University of Illinois at Chicago
Stephanie M Cologna, University of Illinois at Chicago
Stephen J Crocker, University of Connecticut School of Medicine and Dentistry
Maria I Givogri, University of Illinois at Chicago
Mark S Sands, Washington University School of Medicine in St. Louis
Ernesto R Bongarzone, University of Illinois at Chicago

Journal

Molecular therapy

Publication Date

5-5-2021

Volume

29

Issue

5

First Page

1883

Last Page

1902

Document Type

Open Access Publication

DOI

10.1016/j.ymthe.2021.01.026

Rights and Permissions

Heller GJ, Marshall MS, Issa Y, Marshall JN, Nguyen D, Rue E, Pathmasiri KC, Domowicz MS, van Breemen RB, Tai LM, Cologna SM, Crocker SJ, Givogri MI, Sands MS, Bongarzone ER. Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease. Mol Ther. 2021 May 5;29(5):1883-1902. doi: 10.1016/j.ymthe.2021.01.026. Copyright 2021 The Author(s). This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

Recommended Citation

Heller, Gregory J; Marshall, Michael S; Issa, Yazan; Marshall, Jeffrey N; Nguyen, Duc; Rue, Emily; Pathmasiri, Koralege C; Domowicz, Miriam S; van Breemen, Richard B; Tai, Leon M; Cologna, Stephanie M; Crocker, Stephen J; Givogri, Maria I; Sands, Mark S; and Bongarzone, Ernesto R, "Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease." Molecular therapy. 29, 5. 1883 - 1902. (2021).
https://digitalcommons.wustl.edu/open_access_pubs/10797