Therapeutic Benefit of Autophagy Modulation in Pompe Disease.

Lim, Jeong-A; Sun, Baodong; Puertollano, Rosa; Raben, Nina

Lim, Jeong-A; Sun, Baodong; Puertollano, Rosa; Raben, Nina.

Affiliation

Lim JA; Cell Biology and Physiology Center, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD, USA; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Sun B; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Puertollano R; Cell Biology and Physiology Center, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD, USA. Electronic address: puertolr@nhlbi.hih.gov.
Raben N; Cell Biology and Physiology Center, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD, USA. Electronic address: rabenn@mail.nih.gov.

Mol Ther ; 26(7): 1783-1796, 2018 07 05.

Article in En | MEDLINE | ID: mdl-29804932

Subject(s)

Autophagy/physiology; Glycogen Storage Disease Type II/physiopathology; Lysosomes/physiology; Animals; Disease Models, Animal; Enzyme Replacement Therapy/methods; Female; Glycogen/metabolism; Glycogen Storage Disease Type II/metabolism; Lysosomes/metabolism; Male; Mice; Mice, Knockout; Muscle, Skeletal/metabolism; Muscle, Skeletal/physiopathology; TOR Serine-Threonine Kinases/metabolism; Up-Regulation/physiology; alpha-Glucosidases/metabolism

Key words

autophagy; enzyme replacement therapy; lysosomal storage diseases; metabolome; muscle proteostasis

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Autophagy / Glycogen Storage Disease Type II / Lysosomes Limits: Animals Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2018 Type: Article Affiliation country: United States

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