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Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits.
DiFiglia M, Sena-Esteves M, Chase K, Sapp E, Pfister E, Sass M, Yoder J, Reeves P, Pandey RK, Rajeev KG, Manoharan M, Sah DW, Zamore PD, Aronin N. DiFiglia M, et al. Proc Natl Acad Sci U S A. 2007 Oct 23;104(43):17204-9. doi: 10.1073/pnas.0708285104. Epub 2007 Oct 16. Proc Natl Acad Sci U S A. 2007. PMID: 17940007 Free PMC article.
Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors.
Kegel KB, Sapp E, Alexander J, Reeves P, Bleckmann D, Sobin L, Masso N, Valencia A, Jeong H, Krainc D, Palacino J, Curtis D, Kuhn R, Betschart C, Sena-Esteves M, Aronin N, Paganetti P, Difiglia M. Kegel KB, et al. Mol Neurodegener. 2010 Dec 14;5:58. doi: 10.1186/1750-1326-5-58. Mol Neurodegener. 2010. PMID: 21156064 Free PMC article.
163 results