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Journal of Inherited Metabolic Disease
|
June 11, 2026
Validating a Human Cell Model of Null Galactosylceramidase (GALC) Enzyme Activity That Recapitulates Krabbe Disease
Rodrigo T Starosta, Nazia Khatoon, Marie S Roberts, et al.
Gene Therapy
|
March 29, 2013
Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
C D Heldermon, E Y Qin, K K Ohlemiller, et al.
International Journal of Molecular Sciences
|
April 14, 2026
Systemic AAV9 Gene Therapy Mitigates Neuromuscular Junction Degeneration and Muscle Atrophy in a Mouse Model of CLN1 Disease
Ewa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
The Journal of Clinical Investigation
|
April 27, 2023
Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model
Keigo Takahashi, Elizabeth M Eultgen, Sophie H Wang, et al.
Molecular Genetics and Metabolism
|
June 21, 2025
Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases
Pamela Kell, Sonali Mishra, Heather L Gray-Edwards, et al.
The Journal of Clinical Investigation
|
May 17, 2008
Niemann-Pick C1 protects against atherosclerosis in mice via regulation of macrophage intracellular cholesterol trafficking
Jessie R Zhang, Trey Coleman, S Joshua Langmade, et al.
Human Molecular Genetics
|
July 31, 2009
Molecular correlates of axonal and synaptic pathology in mouse models of Batten disease
Catherine Kielar, Thomas M Wishart, Alice Palmer, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
April 24, 2015
Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy
Jacqueline A Hawkins-Salsbury, Lauren Shea, Xuntian Jiang, et al.
Neuromuscular Disorders : NMD
|
July 1, 1997
Gene therapy for murine mucopolysaccharidosis type VII
M S Sands, J H Wolfe, E H Birkenmeier, et al.
Molecular Therapy. Methods & Clinical Development
|
November 22, 2017
A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I
Steven Q Le, Shih-Hsin Kan, Don Clarke, et al.
Page
of 20
Search research articles
Search
Showing results (161-170 of 194) with videos related to
Sort By:
Page
of 20
Journal of Inherited Metabolic Disease
|
June 11, 2026
Validating a Human Cell Model of Null Galactosylceramidase (GALC) Enzyme Activity That Recapitulates Krabbe Disease
Rodrigo T Starosta, Nazia Khatoon, Marie S Roberts, et al.
Gene Therapy
|
March 29, 2013
Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
C D Heldermon, E Y Qin, K K Ohlemiller, et al.
International Journal of Molecular Sciences
|
April 14, 2026
Systemic AAV9 Gene Therapy Mitigates Neuromuscular Junction Degeneration and Muscle Atrophy in a Mouse Model of CLN1 Disease
Ewa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
The Journal of Clinical Investigation
|
April 27, 2023
Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model
Keigo Takahashi, Elizabeth M Eultgen, Sophie H Wang, et al.
Molecular Genetics and Metabolism
|
June 21, 2025
Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases
Pamela Kell, Sonali Mishra, Heather L Gray-Edwards, et al.
The Journal of Clinical Investigation
|
May 17, 2008
Niemann-Pick C1 protects against atherosclerosis in mice via regulation of macrophage intracellular cholesterol trafficking
Jessie R Zhang, Trey Coleman, S Joshua Langmade, et al.
Human Molecular Genetics
|
July 31, 2009
Molecular correlates of axonal and synaptic pathology in mouse models of Batten disease
Catherine Kielar, Thomas M Wishart, Alice Palmer, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
April 24, 2015
Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy
Jacqueline A Hawkins-Salsbury, Lauren Shea, Xuntian Jiang, et al.
Neuromuscular Disorders : NMD
|
July 1, 1997
Gene therapy for murine mucopolysaccharidosis type VII
M S Sands, J H Wolfe, E H Birkenmeier, et al.
Molecular Therapy. Methods & Clinical Development
|
November 22, 2017
A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I
Steven Q Le, Shih-Hsin Kan, Don Clarke, et al.
Page
of 20