Small-molecule activation of lysosomal TRP channels ameliorates Duchenne muscular dystrophy in mouse models

Lysosomal Ion Channels as Decoders of Cellular Signals

(2018) Ping Li et al.   TRENDS IN BIOCHEMICAL SCIENCES

SU9516 Increases α7β1 Integrin and Ameliorates Disease Progression in the mdx Mouse Model of Duchenne Muscular Dystrophy

(2017) Apurva Sarathy et al.   MOLECULAR THERAPY

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

Endo-lysosomal TRP mucolipin-1 channels trigger global ER Ca2+release and Ca2+influx

(2016) Bethan S. Kilpatrick et al.   JOURNAL OF CELL SCIENCE

Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy

(2016) David G. Allen et al.   PHYSIOLOGICAL REVIEWS

MCOLN1 is a ROS sensor in lysosomes that regulates autophagy

(2016) Xiaoli Zhang et al.   Nature Communications

Lysosomal Physiology

(2015) Haoxing Xu et al.   Annual Review of Physiology

Lysosomal calcium signalling regulates autophagy through calcineurin and TFEB

(2015) Diego L. Medina et al.   NATURE CELL BIOLOGY

Up-regulation of lysosomal TRPML1 channels is essential for lysosomal adaptation to nutrient starvation

(2015) Wuyang Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Calcium signaling in membrane repair

(2015) Xiping Cheng et al.   SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY

The intracellular Ca2+ channel MCOLN1 is required for sarcolemma repair to prevent muscular dystrophy

(2014) Xiping Cheng et al.   NATURE MEDICINE

Src-dependent impairment of autophagy by oxidative stress in a mouse model of Duchenne muscular dystrophy

(2014) Rituraj Pal et al.   Nature Communications

Dystrophin deficiency leads to disturbance of LAMP1-vesicle-associated protein secretion

(2013) Stephanie Duguez et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

A TRP Channel in the Lysosome Regulates Large Particle Phagocytosis via Focal Exocytosis

(2013) Mohammad Samie et al.   DEVELOPMENTAL CELL

Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease

(2013) Carmine Spampanato et al.   EMBO Molecular Medicine

Cellular and molecular mechanisms underlying muscular dystrophy

(2013) Fedik Rahimov et al.   JOURNAL OF CELL BIOLOGY

Muscular dystrophies

(2013) Eugenio Mercuri et al.   LANCET

Lipid storage disorders block lysosomal trafficking by inhibiting a TRP channel and lysosomal calcium release

(2012) Dongbiao Shen et al.   Nature Communications

Skeletal muscle weakness due to deficiency of CuZn-superoxide dismutase is associated with loss of functional innervation

(2011) Lisa M. Larkin et al.   AMERICAN JOURNAL OF PHYSIOLOGY-REGULATORY INTEGRATIVE AND COMPARATIVE PHYSIOLOGY

Transcriptional Activation of Lysosomal Exocytosis Promotes Cellular Clearance

(2011) Diego L. Medina et al.   DEVELOPMENTAL CELL

A single 30min treadmill exercise session is suitable for ‘proof-of concept studies’ in adult mdx mice: A comparison of the early consequences of two different treadmill protocols

(2011) Hannah Radley-Crabb et al.   NEUROMUSCULAR DISORDERS

Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders

(2011) Kamel Mamchaoui et al.   Skeletal Muscle

Sildenafil reverses cardiac dysfunction in the mdx mouse model of Duchenne muscular dystrophy

(2010) C. M. Adamo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

PI(3,5)P2 controls membrane trafficking by direct activation of mucolipin Ca2+ release channels in the endolysosome

(2010) Xian-ping Dong et al.   Nature Communications

Mechanisms of brain iron transport: insight into neurodegeneration and CNS disorders

(2009) Eric Mills et al.   Future Medicinal Chemistry

Membrane repair redux: redox of MG53

(2009) Paul McNeil   NATURE CELL BIOLOGY

Repair of injured plasma membrane by rapid Ca2+-dependent endocytosis

(2008) Vincent Idone et al.   JOURNAL OF CELL BIOLOGY

The type IV mucolipidosis-associated protein TRPML1 is an endolysosomal iron release channel

(2008) Xian-Ping Dong et al.   NATURE

MG53 nucleates assembly of cell membrane repair machinery

(2008) Chuanxi Cai et al.   NATURE CELL BIOLOGY