mTOR inhibition alleviates mitochondrial disease in a mouse model of Leigh syndrome
| Autor: | Matt Kaeberlein, Ernst Bernhard Kayser, Melana E. Yanos, Lauren Uhde, Philip G. Morgan, Richard D. Palmiter, Simon C. Johnson, Kelly Oh, Maya Sangesland, Margaret M. Sedensky, Brian M. Wasko, Albert Quintana, Valerie Z. Wall, Peter S. Rabinovitch, Anthony S. Castanza, Fresnida J. Ramos, Arni Gagnidze, Jessica Hui |
|---|---|
| Rok vydání: | 2013 |
| Předmět: |
Mitochondrial Diseases
Mitochondrial disease mTORC1 Mitochondrion Biology Mechanistic Target of Rapamycin Complex 1 Article Mice medicine Animals Molecular Targeted Therapy Leigh disease Mechanistic target of rapamycin PI3K/AKT/mTOR pathway Mice Knockout Sirolimus Multidisciplinary Electron Transport Complex I TOR Serine-Threonine Kinases NDUFS4 Brain medicine.disease Mice Mutant Strains Mitochondria Disease Models Animal Mitochondrial respiratory chain Neuroprotective Agents Biochemistry Multiprotein Complexes biology.protein Cancer research Leigh Disease Glycolysis |
| Zdroj: | Science (New York, N.Y.). 342(6165) |
| ISSN: | 1095-9203 |
| Popis: | More from mTOR Leigh syndrome is a rare, untreatable, inherited neurodegenerative disease in children that is caused by functional disruption of mitochondria, the cell's energy-producing organelles. Johnson et al. (p. 1524 , published online 14 November; see Perspective by Vafai and Mootha ) show that rapamycin, a drug used clinically as an immunosuppressant and for treatment of certain cancers, delayed the onset and progression of neurological symptoms in a mouse model of Leigh syndrome and significantly extended survival of the animals. Rapamycin inhibits the so-called “mTOR” signaling pathway, which is currently under intense study because it plays a contributory role in many common diseases. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|