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Molecular Therapy. Methods & Clinical Development
|
February 28, 2019
Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease
Jeong-A Lim, Haiqing Yi, Fengqin Gao, et al.
Autophagy
|
November 13, 2008
When more is less: excess and deficiency of autophagy coexist in skeletal muscle in Pompe disease
Nina Raben, Rebecca Baum, Cynthia Schreiner, et al.
Plos One
|
December 24, 2010
Fiber type conversion by PGC-1α activates lysosomal and autophagosomal biogenesis in both unaffected and Pompe skeletal muscle
Shoichi Takikita, Cynthia Schreiner, Rebecca Baum, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 1, 2002
Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme
Nina Raben, Tejas Jatkar, Alicia Lee, et al.
Autophagy
|
July 29, 2006
Autophagy and lysosomes in Pompe disease
Tokiko Fukuda, Ashley Roberts, Meghan Ahearn, et al.
EMBO Molecular Medicine
|
January 29, 2017
Modulation of mTOR signaling as a strategy for the treatment of Pompe disease
Jeong-A Lim, Lishu Li, Orian S Shirihai, et al.
Human Molecular Genetics
|
April 28, 2017
Novel degenerative and developmental defects in a zebrafish model of mucolipidosis type IV
Huiqing Li, Wuhong Pei, Sivia Vergarajauregui, et al.
Human Molecular Genetics
|
September 11, 2008
Suppression of autophagy in skeletal muscle uncovers the accumulation of ubiquitinated proteins and their potential role in muscle damage in Pompe disease
Nina Raben, Victoria Hill, Lauren Shea, et al.
Chemical Science
|
October 4, 2021
Chemoenzymatic glycan-selective remodeling of a therapeutic lysosomal enzyme with high-affinity M6P-glycan ligands. Enzyme substrate specificity is the name of the game
Xiao Zhang, Huiying Liu, Naresh Meena, et al.
Science Signaling
|
January 23, 2014
The nutrient-responsive transcription factor TFE3 promotes autophagy, lysosomal biogenesis, and clearance of cellular debris
José A Martina, Heba I Diab, Li Lishu, et al.
Page
of 8
Search research articles
Search
Showing results (31-40 of 72) with videos related to
Sort By:
Page
of 8
Molecular Therapy. Methods & Clinical Development
|
February 28, 2019
Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease
Jeong-A Lim, Haiqing Yi, Fengqin Gao, et al.
Autophagy
|
November 13, 2008
When more is less: excess and deficiency of autophagy coexist in skeletal muscle in Pompe disease
Nina Raben, Rebecca Baum, Cynthia Schreiner, et al.
Plos One
|
December 24, 2010
Fiber type conversion by PGC-1α activates lysosomal and autophagosomal biogenesis in both unaffected and Pompe skeletal muscle
Shoichi Takikita, Cynthia Schreiner, Rebecca Baum, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 1, 2002
Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme
Nina Raben, Tejas Jatkar, Alicia Lee, et al.
Autophagy
|
July 29, 2006
Autophagy and lysosomes in Pompe disease
Tokiko Fukuda, Ashley Roberts, Meghan Ahearn, et al.
EMBO Molecular Medicine
|
January 29, 2017
Modulation of mTOR signaling as a strategy for the treatment of Pompe disease
Jeong-A Lim, Lishu Li, Orian S Shirihai, et al.
Human Molecular Genetics
|
April 28, 2017
Novel degenerative and developmental defects in a zebrafish model of mucolipidosis type IV
Huiqing Li, Wuhong Pei, Sivia Vergarajauregui, et al.
Human Molecular Genetics
|
September 11, 2008
Suppression of autophagy in skeletal muscle uncovers the accumulation of ubiquitinated proteins and their potential role in muscle damage in Pompe disease
Nina Raben, Victoria Hill, Lauren Shea, et al.
Chemical Science
|
October 4, 2021
Chemoenzymatic glycan-selective remodeling of a therapeutic lysosomal enzyme with high-affinity M6P-glycan ligands. Enzyme substrate specificity is the name of the game
Xiao Zhang, Huiying Liu, Naresh Meena, et al.
Science Signaling
|
January 23, 2014
The nutrient-responsive transcription factor TFE3 promotes autophagy, lysosomal biogenesis, and clearance of cellular debris
José A Martina, Heba I Diab, Li Lishu, et al.
Page
of 8