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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 10, 2011
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector
Dawn E Bowles, Scott W J McPhee, Chengwen Li, et al.
Neuroscience
|
May 4, 2020
Intellectual and Developmental Disabilities Research Centers: A Multidisciplinary Approach to Understand the Pathogenesis of Methyl-CpG Binding Protein 2-related Disorders
Michela Fagiolini, Annarita Patrizi, Jocelyn LeBlanc, et al.
Brain : a Journal of Neurology
|
June 11, 2021
Giant axonal neuropathy: cross-sectional analysis of a large natural history cohort
Diana X Bharucha-Goebel, Gina Norato, Dimah Saade, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 14, 2018
Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy
Michael S Marshall, Yazan Issa, Benas Jakubauskas, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 19, 2022
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders
Beverly L Davidson, Guangping Gao, Elizabeth Berry-Kravis, 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.
The New England Journal of Medicine
|
March 20, 2024
Intrathecal Gene Therapy for Giant Axonal Neuropathy
Diana X Bharucha-Goebel, Joshua J Todd, Dimah Saade, et al.
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Search research articles
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Showing results (101-110 of 107) with videos related to
Sort By:
Page
of 11
You have reached the last page of results.
This site can display upto 107 results.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 10, 2011
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector
Dawn E Bowles, Scott W J McPhee, Chengwen Li, et al.
Neuroscience
|
May 4, 2020
Intellectual and Developmental Disabilities Research Centers: A Multidisciplinary Approach to Understand the Pathogenesis of Methyl-CpG Binding Protein 2-related Disorders
Michela Fagiolini, Annarita Patrizi, Jocelyn LeBlanc, et al.
Brain : a Journal of Neurology
|
June 11, 2021
Giant axonal neuropathy: cross-sectional analysis of a large natural history cohort
Diana X Bharucha-Goebel, Gina Norato, Dimah Saade, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 14, 2018
Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy
Michael S Marshall, Yazan Issa, Benas Jakubauskas, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 19, 2022
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders
Beverly L Davidson, Guangping Gao, Elizabeth Berry-Kravis, 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.
The New England Journal of Medicine
|
March 20, 2024
Intrathecal Gene Therapy for Giant Axonal Neuropathy
Diana X Bharucha-Goebel, Joshua J Todd, Dimah Saade, et al.
Page
of 11