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Scientific Reports
|
December 30, 2025
Limited therapeutic efficacy of N-acetyl-L-leucine in a mouse model of CLN1 disease
Ewa A Ziółkowska, Nicole A Pagán Torres, Hsintsung Chen, 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.
The Lancet. Neurology
|
November 25, 2018
Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis
Sara E Mole, Glenn Anderson, Heather A Band, et al.
Acta Neuropathologica Communications
|
October 19, 2017
Glial cells are functionally impaired in juvenile neuronal ceroid lipofuscinosis and detrimental to neurons
Lotta Parviainen, Sybille Dihanich, Greg W Anderson, et al.
Acta Neuropathologica
|
September 11, 2012
Overexpression of human wild-type FUS causes progressive motor neuron degeneration in an age- and dose-dependent fashion
Jacqueline C Mitchell, Philip McGoldrick, Caroline Vance, et al.
FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology
|
June 12, 2015
Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates
Citra N Mattar, Andrew M S Wong, Klemens Hoefer, et al.
Acta Neuropathologica Communications
|
December 22, 2025
Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice
Ewa A Ziółkowska, Letitia L Williams, Matthew J Jansen, et al.
Molecular Genetics and Metabolism
|
June 19, 2012
Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis
Jie Hu, Jui-Yun Lu, Andrew M S Wong, et al.
Biorxiv : the Preprint Server for Biology
|
April 8, 2024
GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice
Keigo Takahashi, Nicholas R Rensing, Elizabeth M Eultgen, et al.
Plos One
|
May 5, 2012
Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB
Fiona L Wilkinson, Rebecca J Holley, Kia J Langford-Smith, et al.
Page
of 15
Search research articles
Search
Showing results (111-120 of 144) with videos related to
Sort By:
Page
of 15
Scientific Reports
|
December 30, 2025
Limited therapeutic efficacy of N-acetyl-L-leucine in a mouse model of CLN1 disease
Ewa A Ziółkowska, Nicole A Pagán Torres, Hsintsung Chen, 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.
The Lancet. Neurology
|
November 25, 2018
Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis
Sara E Mole, Glenn Anderson, Heather A Band, et al.
Acta Neuropathologica Communications
|
October 19, 2017
Glial cells are functionally impaired in juvenile neuronal ceroid lipofuscinosis and detrimental to neurons
Lotta Parviainen, Sybille Dihanich, Greg W Anderson, et al.
Acta Neuropathologica
|
September 11, 2012
Overexpression of human wild-type FUS causes progressive motor neuron degeneration in an age- and dose-dependent fashion
Jacqueline C Mitchell, Philip McGoldrick, Caroline Vance, et al.
FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology
|
June 12, 2015
Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates
Citra N Mattar, Andrew M S Wong, Klemens Hoefer, et al.
Acta Neuropathologica Communications
|
December 22, 2025
Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice
Ewa A Ziółkowska, Letitia L Williams, Matthew J Jansen, et al.
Molecular Genetics and Metabolism
|
June 19, 2012
Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis
Jie Hu, Jui-Yun Lu, Andrew M S Wong, et al.
Biorxiv : the Preprint Server for Biology
|
April 8, 2024
GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice
Keigo Takahashi, Nicholas R Rensing, Elizabeth M Eultgen, et al.
Plos One
|
May 5, 2012
Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB
Fiona L Wilkinson, Rebecca J Holley, Kia J Langford-Smith, et al.
Page
of 15