Timothy M Miller

Summary

Affiliation: Washington University School of Medicine
Country: USA

Publications

  1. doi request reprint Gene-targeted therapies for the central nervous system
    Timothy M Miller
    Neurosciences Department, University of California, San Diego, San Diego, California, USA
    Arch Neurol 65:447-51. 2008
  2. pmc An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study
    Timothy M Miller
    Washington University School of Medicine, St Louis, MO, USA
    Lancet Neurol 12:435-42. 2013
  3. pmc Antisense oligonucleotide therapy for neurodegenerative disease
    Richard A Smith
    Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, California, USA
    J Clin Invest 116:2290-6. 2006
  4. ncbi request reprint Amyotrophic lateral sclerosis and gene therapy
    Timothy M Miller
    Department of Neurosciences, Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093 0670, USA
    Nat Clin Pract Neurol 2:462-3. 2006
  5. pmc TREM2 Variant p.R47H as a Risk Factor for Sporadic Amyotrophic Lateral Sclerosis
    Janet Cady
    Department of Neurology, Washington University School of Medicine, St Louis, Missouri
    JAMA Neurol 71:449-53. 2014
  6. pmc Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis
    Timothy M Miller
    Ludwig Institute for Cancer Research, University of California, San Diego, USA
    Ann Neurol 57:773-6. 2005
  7. ncbi request reprint Progressive spinal axonal degeneration and slowness in ALS2-deficient mice
    Koji Yamanaka
    Ludwig Institute for Cancer Research and Department of Medicine and Neurosciences, University of California, San Diego, La Jolla, 92093 0670, USA
    Ann Neurol 60:95-104. 2006
  8. pmc ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import
    Quan Li
    Department of Neurology, Hope Center for Neurological Disorders, The Washington University School of Medicine, St Louis, MO 63110, USA
    Proc Natl Acad Sci U S A 107:21146-51. 2010
  9. pmc Canine degenerative myelopathy: biochemical characterization of superoxide dismutase 1 in the first naturally occurring non-human amyotrophic lateral sclerosis model
    Matthew J Crisp
    Department of Neurology, Washington University School of Medicine, St Louis, MO 63110, USA
    Exp Neurol 248:1-9. 2013
  10. pmc TDP-43 mutant transgenic mice develop features of ALS and frontotemporal lobar degeneration
    Iga Wegorzewska
    Department of Neurology and Hope Center for Neurological Diseases, Washington University School of Medicine, St Louis, MO 63110, USA
    Proc Natl Acad Sci U S A 106:18809-14. 2009

Collaborators

Detail Information

Publications25

  1. doi request reprint Gene-targeted therapies for the central nervous system
    Timothy M Miller
    Neurosciences Department, University of California, San Diego, San Diego, California, USA
    Arch Neurol 65:447-51. 2008
  2. pmc An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study
    Timothy M Miller
    Washington University School of Medicine, St Louis, MO, USA
    Lancet Neurol 12:435-42. 2013
    ..We aimed to assess the safety, tolerability, and pharmacokinetics of ISIS 333611 after intrathecal administration in patients with SOD1-related familial amyotrophic lateral sclerosis...
  3. pmc Antisense oligonucleotide therapy for neurodegenerative disease
    Richard A Smith
    Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, California, USA
    J Clin Invest 116:2290-6. 2006
    ..This suggests that direct delivery of antisense oligonucleotides could be an effective, dosage-regulatable means of treating neurodegenerative diseases, including ALS, where appropriate target proteins are known...
  4. ncbi request reprint Amyotrophic lateral sclerosis and gene therapy
    Timothy M Miller
    Department of Neurosciences, Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093 0670, USA
    Nat Clin Pract Neurol 2:462-3. 2006
  5. pmc TREM2 Variant p.R47H as a Risk Factor for Sporadic Amyotrophic Lateral Sclerosis
    Janet Cady
    Department of Neurology, Washington University School of Medicine, St Louis, Missouri
    JAMA Neurol 71:449-53. 2014
    ..To our knowledge, these findings identify the first genetic influence on neuroinflammation in ALS and highlight the TREM2 signaling pathway as a therapeutic target in ALS and other neurodegenerative diseases. ..
  6. pmc Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis
    Timothy M Miller
    Ludwig Institute for Cancer Research, University of California, San Diego, USA
    Ann Neurol 57:773-6. 2005
    ....
  7. ncbi request reprint Progressive spinal axonal degeneration and slowness in ALS2-deficient mice
    Koji Yamanaka
    Ludwig Institute for Cancer Research and Department of Medicine and Neurosciences, University of California, San Diego, La Jolla, 92093 0670, USA
    Ann Neurol 60:95-104. 2006
    ..The goal of this study was to elucidate how the motor system is affected by the deletion of ALS2...
  8. pmc ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import
    Quan Li
    Department of Neurology, Hope Center for Neurological Disorders, The Washington University School of Medicine, St Louis, MO 63110, USA
    Proc Natl Acad Sci U S A 107:21146-51. 2010
    ..Thus, altered mitochondrial protein content accompanied by selective decreases in protein import into spinal cord mitochondria comprises part of the mitochondrial damage arising from mutant SOD1...
  9. pmc Canine degenerative myelopathy: biochemical characterization of superoxide dismutase 1 in the first naturally occurring non-human amyotrophic lateral sclerosis model
    Matthew J Crisp
    Department of Neurology, Washington University School of Medicine, St Louis, MO 63110, USA
    Exp Neurol 248:1-9. 2013
    ..Our findings lend strong biochemical support to the toxic role of SOD1 in canine degenerative myelopathy and establish close parallels for the role mutant SOD1 plays in both canine and human disorders. ..
  10. pmc TDP-43 mutant transgenic mice develop features of ALS and frontotemporal lobar degeneration
    Iga Wegorzewska
    Department of Neurology and Hope Center for Neurological Diseases, Washington University School of Medicine, St Louis, MO 63110, USA
    Proc Natl Acad Sci U S A 106:18809-14. 2009
    ....
  11. doi request reprint Familial ALS with extreme phenotypic variability due to the I113T SOD1 mutation
    Glenn Lopate
    Washington University School of Medicine, Department of Neurology, Saint Louis, Missouri 63110, USA
    Amyotroph Lateral Scler 11:232-6. 2010
    ..This family highlights the extreme variability in age of onset, clinical manifestations, disease progression and penetrance due to the I113T SOD1 mutation...
  12. ncbi request reprint Distinct tau prion strains propagate in cells and mice and define different tauopathies
    David W Sanders
    Department of Neurology, Washington University in St Louis, St Louis, MO 63105, USA
    Neuron 82:1271-88. 2014
    ..Tau thus demonstrates essential characteristics of a prion. This might explain the phenotypic diversity of tauopathies and could enable more effective diagnosis and therapy...
  13. pmc SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy
    Leah Winer
    Department of Neurology, Washington University, St Louis, Missouri 63110, USA
    JAMA Neurol 70:201-7. 2013
    ..Therapies designed to decrease the level of SOD1 are currently in a clinical trial for patients with superoxide dismutase (SOD1)-linked familial amyotrophic lateral sclerosis (ALS)...
  14. ncbi request reprint Muscle cramps
    Timothy M Miller
    Department of Neurosciences, University of California, San Diego, 9500 Gilman Drive, La Jolla, California 92093 0670, USA
    Muscle Nerve 32:431-42. 2005
    ..Patients will benefit from further studies to better define the pathophysiology of muscle cramps and to find more effective medications with fewer side-effects...
  15. ncbi request reprint Medicine. Treating neurodegenerative diseases with antibiotics
    Timothy M Miller
    Ludwig Institute for Cancer Research and the Department of Medicine and Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA
    Science 307:361-2. 2005
  16. ncbi request reprint Should the Babinski sign be part of the routine neurologic examination?
    Timothy M Miller
    Department of Neurology, University of California, San Francisco, CA, USA
    Neurology 65:1165-8. 2005
    ..A less well-known sign of upper motor neuron dysfunction, decreased speed of foot tapping, also has not been carefully evaluated. Scientific evaluation of findings of the physical examination is crucial in directing busy clinicians...
  17. pmc Antisense oligonucleotides: treating neurodegeneration at the level of RNA
    Sarah L DeVos
    Department of Neurology, Washington University in St Louis School of Medicine, St Louis, MO 63110, USA
    Neurotherapeutics 10:486-97. 2013
    ..Although still early in development, translating ASOs into human patients for neurodegeneration appears promising. ..
  18. pmc Antisense reduction of tau in adult mice protects against seizures
    Sarah L DeVos
    Department of Neurology, Hope Center for Neurological Disorders, Washington University, St Louis, Missouri 63110, USA
    J Neurosci 33:12887-97. 2013
    ....
  19. pmc Heparan sulfate proteoglycans mediate internalization and propagation of specific proteopathic seeds
    Brandon B Holmes
    Department of Neurology, Washington University School of Medicine, St Louis, MO 63110, USA
    Proc Natl Acad Sci U S A 110:E3138-47. 2013
    ..Finally, uptake and seeding by α-synuclein fibrils, but not huntingtin fibrils, occurs by the same mechanism as tau. This work suggests a unifying mechanism of cell uptake and propagation for tauopathy and synucleinopathy. ..
  20. ncbi request reprint Management of amyotrophic lateral sclerosis
    Taha Bali
    Neuromuscular Disease Division, Washington University School of Medicine, St Louis, USA
    Mo Med 110:417-21. 2013
    ..The diagnosis is made clinically, supported by electrodiagnostic testing. Although medications are limited, careful attention to breathing, nutrition, and patient mobility can have a major, positive impact on the course of the disease...
  21. ncbi request reprint Differential diagnosis of myotonic disorders
    Timothy M Miller
    Neurology Department, Washington University, St Louis, MI, USA
    Muscle Nerve 37:293-9. 2008
    ..Acid maltase deficiency often produces myotonic potentials without clinical evidence of myotonia or paramyotonia. The differential diagnosis of these myotonic disorders is discussed...
  22. ncbi request reprint Toxicity of familial ALS-linked SOD1 mutants from selective recruitment to spinal mitochondria
    Jian Liu
    Ludwig Institute for Cancer Research, Department of Neurosciences, Medicine, and Cellular and Molecular Medicine, University of California, San Diego, 9500 Gilman Drive, La Jolla, CA 92093, USA
    Neuron 43:5-17. 2004
    ..These findings implicate damage from action of spinal cord-specific factors that recruit mutant SOD1 to spinal mitochondria as the basis for their selective toxicity in ALS...
  23. ncbi request reprint Proposed modification to data analysis for statistical motor unit number estimate
    Timothy M Miller
    Department of Neurology, University of California San Francisco, San Francisco, California, USA
    Muscle Nerve 29:700-6. 2004
    ....
  24. pmc Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis
    Timothy M Miller
    Ludwig Institute for Cancer Research, La Jolla, CA 92093, USA
    Proc Natl Acad Sci U S A 103:19546-51. 2006
    ..Thus, SOD1-mutant-mediated damage within muscles is not a significant contributor to non-cell-autonomous pathogenesis in ALS, and enhancing muscle mass and strength provides no benefit in slowing disease onset or progression...
  25. ncbi request reprint Has gene therapy for ALS arrived?
    Timothy M Miller
    Nat Med 9:1256-7. 2003