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An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study.
Miller TM, Pestronk A, David W, Rothstein J, Simpson E, Appel SH, Andres PL, Mahoney K, Allred P, Alexander K, Ostrow LW, Schoenfeld D, Macklin EA, Norris DA, Manousakis G, Crisp M, Smith R, Bennett CF, Bishop KM, Cudkowicz ME. Miller TM, et al. Lancet Neurol. 2013 May;12(5):435-42. doi: 10.1016/S1474-4422(13)70061-9. Epub 2013 Mar 29. Lancet Neurol. 2013. PMID: 23541756 Free PMC article. Clinical Trial.
Antisense oligonucleotide therapy for neurodegenerative disease.
Smith RA, Miller TM, Yamanaka K, Monia BP, Condon TP, Hung G, Lobsiger CS, Ward CM, McAlonis-Downes M, Wei H, Wancewicz EV, Bennett CF, Cleveland DW. Smith RA, et al. Among authors: miller tm. J Clin Invest. 2006 Aug;116(8):2290-6. doi: 10.1172/JCI25424. Epub 2006 Jul 27. J Clin Invest. 2006. PMID: 16878173 Free PMC article.
Amyotrophic lateral sclerosis and gene therapy.
Miller TM, Smith RA, Cleveland DW. Miller TM, et al. Nat Clin Pract Neurol. 2006 Sep;2(9):462-3. doi: 10.1038/ncpneuro0270. Nat Clin Pract Neurol. 2006. PMID: 16932606 Review. No abstract available.
Gene-targeted therapies for the central nervous system.
Miller TM, Smith RA, Kordasiewicz H, Kaspar BK. Miller TM, et al. Arch Neurol. 2008 Apr;65(4):447-51. doi: 10.1001/archneur.65.4.nnr70007. Epub 2008 Feb 11. Arch Neurol. 2008. PMID: 18268183 Review. No abstract available.
ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import.
Li Q, Vande Velde C, Israelson A, Xie J, Bailey AO, Dong MQ, Chun SJ, Roy T, Winer L, Yates JR, Capaldi RA, Cleveland DW, Miller TM. Li Q, et al. Among authors: miller tm. Proc Natl Acad Sci U S A. 2010 Dec 7;107(49):21146-51. doi: 10.1073/pnas.1014862107. Epub 2010 Nov 15. Proc Natl Acad Sci U S A. 2010. PMID: 21078990 Free PMC article.
Antisense reduction of tau in adult mice protects against seizures.
DeVos SL, Goncharoff DK, Chen G, Kebodeaux CS, Yamada K, Stewart FR, Schuler DR, Maloney SE, Wozniak DF, Rigo F, Bennett CF, Cirrito JR, Holtzman DM, Miller TM. DeVos SL, et al. Among authors: miller tm. J Neurosci. 2013 Jul 31;33(31):12887-97. doi: 10.1523/JNEUROSCI.2107-13.2013. J Neurosci. 2013. PMID: 23904623 Free PMC article.
283 results