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Molecular Therapy. Methods & Clinical Development
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June 9, 2015
A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I
Daniel C Mendez, Alexander E Stover, Anthony D Rangel, et al.
Molecular Genetics and Metabolism
|
July 27, 2010
Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I
Patricia I Dickson, Stephen Hanson, Michael F McEntee, et al.
Plos Pathogens
|
April 15, 2016
The Epstein-Barr Virus Glycoprotein gp150 Forms an Immune-Evasive Glycan Shield at the Surface of Infected Cells
Anna M Gram, Timo Oosenbrug, Marthe F S Lindenbergh, et al.
The Journal of Biological Chemistry
|
June 24, 2022
Impaired mitophagy in Sanfilippo a mice causes hypertriglyceridemia and brown adipose tissue activation
Miguel Tillo, William C Lamanna, Chrissa A Dwyer, et al.
Molecular Genetics and Metabolism
|
November 25, 2015
Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction
Priya S Kishnani, Patricia I Dickson, Laurie Muldowney, et al.
Science Translational Medicine
|
January 15, 2025
Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models
Ewa A Ziółkowska, Matthew J Jansen, Letitia L Williams, et al.
Molecular Genetics and Metabolism
|
December 24, 2010
Research challenges in central nervous system manifestations of inborn errors of metabolism
P I Dickson, A R Pariser, S C Groft, et al.
Science Translational Medicine
|
December 3, 2010
Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I
Ashley D Dierenfeld, Michael F McEntee, Carole A Vogler, et al.
The Journal of Clinical Investigation
|
August 30, 2022
Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep
Hemanth R Nelvagal, Samantha L Eaton, Sophie H Wang, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
October 1, 2014
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB
Shih-Hsin Kan, Mika Aoyagi-Scharber, Steven Q Le, et al.
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Search research articles
Search
Showing results (81-90 of 93) with videos related to
Sort By:
Page
of 10
Molecular Therapy. Methods & Clinical Development
|
June 9, 2015
A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I
Daniel C Mendez, Alexander E Stover, Anthony D Rangel, et al.
Molecular Genetics and Metabolism
|
July 27, 2010
Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I
Patricia I Dickson, Stephen Hanson, Michael F McEntee, et al.
Plos Pathogens
|
April 15, 2016
The Epstein-Barr Virus Glycoprotein gp150 Forms an Immune-Evasive Glycan Shield at the Surface of Infected Cells
Anna M Gram, Timo Oosenbrug, Marthe F S Lindenbergh, et al.
The Journal of Biological Chemistry
|
June 24, 2022
Impaired mitophagy in Sanfilippo a mice causes hypertriglyceridemia and brown adipose tissue activation
Miguel Tillo, William C Lamanna, Chrissa A Dwyer, et al.
Molecular Genetics and Metabolism
|
November 25, 2015
Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction
Priya S Kishnani, Patricia I Dickson, Laurie Muldowney, et al.
Science Translational Medicine
|
January 15, 2025
Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models
Ewa A Ziółkowska, Matthew J Jansen, Letitia L Williams, et al.
Molecular Genetics and Metabolism
|
December 24, 2010
Research challenges in central nervous system manifestations of inborn errors of metabolism
P I Dickson, A R Pariser, S C Groft, et al.
Science Translational Medicine
|
December 3, 2010
Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I
Ashley D Dierenfeld, Michael F McEntee, Carole A Vogler, et al.
The Journal of Clinical Investigation
|
August 30, 2022
Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep
Hemanth R Nelvagal, Samantha L Eaton, Sophie H Wang, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
October 1, 2014
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB
Shih-Hsin Kan, Mika Aoyagi-Scharber, Steven Q Le, et al.
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of 10