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Ganglioside GM1 induces phosphorylation of mutant huntingtin and restores normal motor behavior in Huntington disease mice.
| Content Provider | Semantic Scholar |
|---|---|
| Author | Pardo, Alba Di Maglione, Vittorio Alpaugh, Melanie Horkey, Melanie Atwal, Randy Singh Sassone, Jenny Ciammola, Andrea Steffan, Joan S. Fouad, Karim Truant, Ray Sipione, Simonetta |
| Copyright Year | 2012 |
| Abstract | Huntington disease (HD) is a progressive neurodegenerative monogenic disorder caused by expansion of a polyglutamine stretch in the huntingtin (Htt) protein. Mutant huntingtin triggers neural dysfunction and death, mainly in the corpus striatum and cerebral cortex, resulting in pathognomonic motor symptoms, as well as cognitive and psychiatric decline. Currently, there is no effective treatment for HD. We report that intraventricular infusion of ganglioside GM1 induces phosphorylation of mutant huntingtin at specific serine amino acid residues that attenuate huntingtin toxicity, and restores normal motor function in already symptomatic HD mice. Thus, our studies have identified a potential therapy for HD that targets a posttranslational modification of mutant huntingtin with critical effects on disease pathogenesis. |
| File Format | PDF HTM / HTML |
| DOI | 10.1073/pnas.1114502109 |
| PubMed reference number | 22331905 |
| Journal | Medline |
| Volume Number | 109 |
| Issue Number | 9 |
| Alternate Webpage(s) | http://www.pnas.org/content/109/9/3528.full.pdf?with-ds=yes |
| Alternate Webpage(s) | http://www.pnas.org/content/109/9/3528.full.pdf |
| Alternate Webpage(s) | https://doi.org/10.1073/pnas.1114502109 |
| Journal | Proceedings of the National Academy of Sciences of the United States of America |
| Language | English |
| Access Restriction | Open |
| Content Type | Text |
| Resource Type | Article |
