Timothy M. Miller, M.D., Ph.D.

Neuromuscular Disorders

Neurosciences Program
Molecular Cell Biology Program
Biochemistry, Biophysics, and Structural Biology Program

  • 314-362-8169

  • 314-362-8168

  • 314-362-3279

  • 8111

  • 117 Biotechnology Building

  • millert@neuro.wustl.edu

  • http://millerlab.wustl.edu/

  • ALS (amyotrophic lateral sclerosis), SOD1, TDP-43, C9ORF72, miRNA, Tau, Dementia, Neurodegeneration, Antisense oligonucleotides

  • Mechanisms of neurodegeneration and targeted therapies for neurodegenerative diseases

Research Abstract:

The Miller Laboratory is dedicated to understanding neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS) and dementias, in order to develop new, effective, and safe treatments. Part of the Department of Neurology at Washington University School of Medicine in St. Louis, the Miller Laboratory is headed by Timothy M. Miller, MD, PhD, the David Clayson Professor of Neurology. Dr. Miller is a national leader in translational neuroscience and new therapeutic approaches for neurodegenerative diseases.

Selected Publications:

1. 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. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol 2013;12(5):435-442. PMCID: 3712285

2. Crisp MJ, Mawuenyega KG, Patterson BW, Reddy NC, Chott R, Self WK, Weihl CC, Jockel-Balsarotti J, Varadhachary AS, Bucelli RC, Yarasheski KE, Bateman RJ, Miller TM. In vivo kinetic approach reveals slow SOD1 turnover in the CNS. The Journal of Clinical Investigation 2015;125(7):2772-2780. PMID: 26075819

3. Cady J, Koval ED, Benitez BA, Zaidman C, Jockel-Balsarotti J, Allred P, Baloh RH, Ravits J, Simpson E, Appel SH, Pestronk A, Goate AM, Miller TM, Cruchaga C, Harms MB. TREM2 variant p.R47H as a risk factor for sporadic amyotrophic lateral sclerosis. JAMA Neurol 2014;71(4):449-453. PMCID: 4087113

4. Koval ED, Shaner C, Zhang P, du Maine X, Fischer K, Tay J, Chau BN, Wu GF, Miller TM. Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice. Hum Mol Genet 2013;22(20):4127-4135. PMCID: 3781640

5. Winer L, Srinivasan D, Chun S, Lacomis D, Jaffa M, Fagan A, Holtzman DM, Wancewicz E, Bennett CF, Bowser R, Cudkowicz M, Miller TM. SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy. JAMA Neurol 2013;70(2):201-207. PMCID: 3812918

6. 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. Antisense reduction of tau in adult mice protects against seizures. J Neurosci 2013;33(31):12887-12897. PMCID: 3728694

7. 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. ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import. Proc Natl Acad Sci U S A 2010;107(49):21146-21151. PMCID: 3000256

8. 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. Antisense oligonucleotide therapy for neurodegenerative disease. J Clin Invest 2006;116(8):2290-2296

(most up-to-date pubs and full PDFs at http://millerlab.wustl.edu/publications.html)

Last Updated: 2/19/2016 9:53:21 AM

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