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Human Gene Therapy
|
January 11, 2018
AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)
Allison M Bradbury, Mohammed A Rafi, Jessica H Bagel, et al.
The Journal of Clinical Endocrinology and Metabolism
|
December 15, 2015
A Diet Rich in Medium-Chain Fatty Acids Improves Systolic Function and Alters the Lipidomic Profile in Patients With Type 2 Diabetes: A Pilot Study
Sophia Airhart, W Todd Cade, Hui Jiang, et al.
Science Translational Medicine
|
February 27, 2015
Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease
Charles H Vite, Jessica H Bagel, Gary P Swain, et al.
The Journal of Biological Chemistry
|
October 5, 2012
δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders
Miao Xu, Ke Liu, Manju Swaroop, et al.
Molecular Genetics and Metabolism
|
November 28, 2017
Glucocerebrosidase haploinsufficiency in A53T α-synuclein mice impacts disease onset and course
Nahid Tayebi, Loukia Parisiadou, Bahafta Berhe, et al.
The Journal of Clinical Investigation
|
August 11, 2020
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy
Allison M Bradbury, Jessica H Bagel, Duc Nguyen, et al.
Science Translational Medicine
|
May 6, 2016
Development of a bile acid-based newborn screen for Niemann-Pick disease type C
Xuntian Jiang, Rohini Sidhu, Laurel Mydock-McGrane, et al.
Disease Models & Mechanisms
|
August 3, 2016
A new glucocerebrosidase-deficient neuronal cell model provides a tool to probe pathophysiology and therapeutics for Gaucher disease
Wendy Westbroek, Matthew Nguyen, Marina Siebert, et al.
Journal of Inherited Metabolic Disease
|
January 31, 2023
Elevated oxysterol and N-palmitoyl-O-phosphocholineserine levels in congenital disorders of glycosylation
An N Dang Do, Irene J Chang, Xutian Jiang, et al.
Wellcome Open Research
|
December 24, 2016
Pathogenic mycobacteria achieve cellular persistence by inhibiting the Niemann-Pick Type C disease cellular pathway
Paul Fineran, Emyr Lloyd-Evans, Nathan A Lack, et al.
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of 15
Search research articles
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Showing results (131-140 of 147) with videos related to
Sort By:
Page
of 15
Human Gene Therapy
|
January 11, 2018
AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)
Allison M Bradbury, Mohammed A Rafi, Jessica H Bagel, et al.
The Journal of Clinical Endocrinology and Metabolism
|
December 15, 2015
A Diet Rich in Medium-Chain Fatty Acids Improves Systolic Function and Alters the Lipidomic Profile in Patients With Type 2 Diabetes: A Pilot Study
Sophia Airhart, W Todd Cade, Hui Jiang, et al.
Science Translational Medicine
|
February 27, 2015
Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease
Charles H Vite, Jessica H Bagel, Gary P Swain, et al.
The Journal of Biological Chemistry
|
October 5, 2012
δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders
Miao Xu, Ke Liu, Manju Swaroop, et al.
Molecular Genetics and Metabolism
|
November 28, 2017
Glucocerebrosidase haploinsufficiency in A53T α-synuclein mice impacts disease onset and course
Nahid Tayebi, Loukia Parisiadou, Bahafta Berhe, et al.
The Journal of Clinical Investigation
|
August 11, 2020
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy
Allison M Bradbury, Jessica H Bagel, Duc Nguyen, et al.
Science Translational Medicine
|
May 6, 2016
Development of a bile acid-based newborn screen for Niemann-Pick disease type C
Xuntian Jiang, Rohini Sidhu, Laurel Mydock-McGrane, et al.
Disease Models & Mechanisms
|
August 3, 2016
A new glucocerebrosidase-deficient neuronal cell model provides a tool to probe pathophysiology and therapeutics for Gaucher disease
Wendy Westbroek, Matthew Nguyen, Marina Siebert, et al.
Journal of Inherited Metabolic Disease
|
January 31, 2023
Elevated oxysterol and N-palmitoyl-O-phosphocholineserine levels in congenital disorders of glycosylation
An N Dang Do, Irene J Chang, Xutian Jiang, et al.
Wellcome Open Research
|
December 24, 2016
Pathogenic mycobacteria achieve cellular persistence by inhibiting the Niemann-Pick Type C disease cellular pathway
Paul Fineran, Emyr Lloyd-Evans, Nathan A Lack, et al.
Page
of 15