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The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
July 8, 2011
Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy
Adarsh S Reddy, Joong H Kim, Jacqueline A Hawkins-Salsbury, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 3, 2021
Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease
Yedda Li, Christopher A Miller, Lauren K Shea, et al.
JCI Insight
|
August 21, 2025
GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice
Keigo Takahashi, Nicholas R Rensing, Elizabeth M Eultgen, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 28, 2021
Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease
Gregory J Heller, Michael S Marshall, Yazan Issa, et al.
Acta Neuropathologica Communications
|
July 23, 2025
Gene therapy ameliorates neuromuscular pathology in CLN3 disease
Ewa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
Scientific Reports
|
July 15, 2021
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor
Michael C Babcock, Christina R Mikulka, Bing Wang, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
September 19, 2019
Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target
Yedda Li, Yue Xu, Bruno A Benitez, et al.
Biorxiv : the Preprint Server for Biology
|
November 28, 2024
Haploinsufficiency of lysosomal enzyme genes in Alzheimer's disease
Bruno A Benitez, Clare E Wallace, Maulikkumar Patel, et al.
Biorxiv : the Preprint Server for Biology
|
February 20, 2025
Identifying and treating CLN3 disease outside the central nervous system
Ewa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
The Journal of Clinical Investigation
|
August 11, 2020
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy
Allison M Bradbury, Jessica H Bagel, Duc Nguyen, et al.
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Search research articles
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Showing results (91-100 of 105) with videos related to
Sort By:
Page
of 11
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
July 8, 2011
Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy
Adarsh S Reddy, Joong H Kim, Jacqueline A Hawkins-Salsbury, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 3, 2021
Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease
Yedda Li, Christopher A Miller, Lauren K Shea, et al.
JCI Insight
|
August 21, 2025
GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice
Keigo Takahashi, Nicholas R Rensing, Elizabeth M Eultgen, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 28, 2021
Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease
Gregory J Heller, Michael S Marshall, Yazan Issa, et al.
Acta Neuropathologica Communications
|
July 23, 2025
Gene therapy ameliorates neuromuscular pathology in CLN3 disease
Ewa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
Scientific Reports
|
July 15, 2021
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor
Michael C Babcock, Christina R Mikulka, Bing Wang, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
September 19, 2019
Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target
Yedda Li, Yue Xu, Bruno A Benitez, et al.
Biorxiv : the Preprint Server for Biology
|
November 28, 2024
Haploinsufficiency of lysosomal enzyme genes in Alzheimer's disease
Bruno A Benitez, Clare E Wallace, Maulikkumar Patel, et al.
Biorxiv : the Preprint Server for Biology
|
February 20, 2025
Identifying and treating CLN3 disease outside the central nervous system
Ewa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
The Journal of Clinical Investigation
|
August 11, 2020
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy
Allison M Bradbury, Jessica H Bagel, Duc Nguyen, et al.
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of 11