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Molecular Genetics and Metabolism
|
June 18, 2010
Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease
Karen M Ashe, Kristin M Taylor, Qiuming Chu, et al.
American Journal of Human Genetics
|
March 19, 2019
Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates
Xuling Zhu, Ling Yin, Matt Theisen, et al.
Journal of Hepatology
|
December 19, 2020
Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4<sup>-/-</sup> mouse model of PFIC3
Guangyan Wei, Jingsong Cao, Pinzhu Huang, et al.
American Journal of Human Genetics
|
August 22, 2023
GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals
Menno Ter Huurne, Benjamin L Parker, Ning Qing Liu, et al.
Science Translational Medicine
|
January 10, 2024
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria
Sonam Gurung, Oskar Vilhelmsson Timmermand, Dany Perocheau, et al.
Nature Communications
|
May 26, 2021
mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease
Jingsong Cao, Minjung Choi, Eleonora Guadagnin, et al.
Journal of Inherited Metabolic Disease
|
December 4, 2023
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria
Sonam Gurung, Saketh Karamched, Dany Perocheau, et al.
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of 2
Search research articles
Search
Showing results (11-20 of 17) with videos related to
Sort By:
Page
of 2
You have reached the last page of results.
This site can display upto 17 results.
Molecular Genetics and Metabolism
|
June 18, 2010
Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease
Karen M Ashe, Kristin M Taylor, Qiuming Chu, et al.
American Journal of Human Genetics
|
March 19, 2019
Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates
Xuling Zhu, Ling Yin, Matt Theisen, et al.
Journal of Hepatology
|
December 19, 2020
Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4<sup>-/-</sup> mouse model of PFIC3
Guangyan Wei, Jingsong Cao, Pinzhu Huang, et al.
American Journal of Human Genetics
|
August 22, 2023
GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals
Menno Ter Huurne, Benjamin L Parker, Ning Qing Liu, et al.
Science Translational Medicine
|
January 10, 2024
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria
Sonam Gurung, Oskar Vilhelmsson Timmermand, Dany Perocheau, et al.
Nature Communications
|
May 26, 2021
mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease
Jingsong Cao, Minjung Choi, Eleonora Guadagnin, et al.
Journal of Inherited Metabolic Disease
|
December 4, 2023
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria
Sonam Gurung, Saketh Karamched, Dany Perocheau, et al.
Page
of 2