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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 1, 2014
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates
Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer, et al.
Annals of Clinical and Translational Neurology
|
February 11, 2014
Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept
W David Arnold, Paul N Porensky, Vicki L McGovern, et al.
Glia
|
October 10, 2022
CuATSM effectively ameliorates ALS patient astrocyte-mediated motor neuron toxicity in human in vitro models of amyotrophic lateral sclerosis
Cassandra N Dennys, Florence Roussel, Rochelle Rodrigo, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 21, 2011
Rapid and efficient generation of functional motor neurons from human pluripotent stem cells using gene delivered transcription factor codes
Mark E Hester, Matthew J Murtha, SungWon Song, et al.
Thyroid : Official Journal of the American Thyroid Association
|
March 30, 2022
AAV9-MCT8 Delivery at Juvenile Stage Ameliorates Neurological and Behavioral Deficits in a Mouse Model of MCT8-Deficiency
Xiao-Hui Liao, Pablo Avalos, Oksana Shelest, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 4, 2020
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease
Tyler B Johnson, Katherine A White, Jon J Brudvig, et al.
Human Molecular Genetics
|
April 13, 2018
Mutations in glycyl-tRNA synthetase impair mitochondrial metabolism in neurons
Veronika Boczonadi, Kathrin Meyer, Humberto Gonczarowska-Jorge, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
October 1, 2016
Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism
Laura Ferraiuolo, Kathrin Meyer, Thomas W Sherwood, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
January 1, 2014
Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS
Kathrin Meyer, Laura Ferraiuolo, Carlos J Miranda, et al.
Molecular Therapy. Methods & Clinical Development
|
March 5, 2021
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
Katherine A White, Hemanth R Nelvagal, Timothy A Poole, et al.
Page
of 7
Search research articles
Search
Showing results (41-50 of 61) with videos related to
Sort By:
Page
of 7
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 1, 2014
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates
Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer, et al.
Annals of Clinical and Translational Neurology
|
February 11, 2014
Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept
W David Arnold, Paul N Porensky, Vicki L McGovern, et al.
Glia
|
October 10, 2022
CuATSM effectively ameliorates ALS patient astrocyte-mediated motor neuron toxicity in human in vitro models of amyotrophic lateral sclerosis
Cassandra N Dennys, Florence Roussel, Rochelle Rodrigo, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 21, 2011
Rapid and efficient generation of functional motor neurons from human pluripotent stem cells using gene delivered transcription factor codes
Mark E Hester, Matthew J Murtha, SungWon Song, et al.
Thyroid : Official Journal of the American Thyroid Association
|
March 30, 2022
AAV9-MCT8 Delivery at Juvenile Stage Ameliorates Neurological and Behavioral Deficits in a Mouse Model of MCT8-Deficiency
Xiao-Hui Liao, Pablo Avalos, Oksana Shelest, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 4, 2020
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease
Tyler B Johnson, Katherine A White, Jon J Brudvig, et al.
Human Molecular Genetics
|
April 13, 2018
Mutations in glycyl-tRNA synthetase impair mitochondrial metabolism in neurons
Veronika Boczonadi, Kathrin Meyer, Humberto Gonczarowska-Jorge, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
October 1, 2016
Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism
Laura Ferraiuolo, Kathrin Meyer, Thomas W Sherwood, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
January 1, 2014
Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS
Kathrin Meyer, Laura Ferraiuolo, Carlos J Miranda, et al.
Molecular Therapy. Methods & Clinical Development
|
March 5, 2021
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
Katherine A White, Hemanth R Nelvagal, Timothy A Poole, et al.
Page
of 7