Therapeutic Benefit of Autophagy Modulation in Pompe Disease.

Lim, Jeong-A

Therapeutic Benefit of Autophagy Modulation in Pompe Disease. [electronic resource] - Molecular therapy : the journal of the American Society of Gene Therapy 07 2018 - 1783-1796 p. digital

Publication Type: Journal Article; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't

1525-0024

10.1016/j.ymthe.2018.04.025 doi


Animals
Autophagy--physiology
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
TOR Serine-Threonine Kinases--metabolism
Up-Regulation--physiology
alpha-Glucosidases--metabolism