Brief Communication

Sustained improvement of spinal muscular atrophy mice treated with trichostatin a plus nutrition

  1. Heather L. Narver VMD1,
  2. Lingling Kong PhD2,
  3. Barrington G. Burnett PhD3,
  4. Dong W. Choe BA2,
  5. Marta Bosch-Marcé PhD2,
  6. Addis A. Taye BS3,
  7. Michael A. Eckhaus VMD4,
  8. Charlotte J. Sumner MD2,*

Article first published online: 25 JUL 2008

DOI: 10.1002/ana.21449

Issue

Annals of Neurology

Annals of Neurology

Volume 64, Issue 4, pages 465–470, October 2008

Additional Information

How to Cite

Narver, H. L., Kong, L., Burnett, B. G., Choe, D. W., Bosch-Marcé, M., Taye, A. A., Eckhaus, M. A. and Sumner, C. J. (2008), Sustained improvement of spinal muscular atrophy mice treated with trichostatin a plus nutrition. Annals of Neurology, 64: 465–470. doi: 10.1002/ana.21449

Author Information

  1. 1

    Animal Care Division, National Institute of Neurological Disorders and Stroke, National Institute of Health, Johns Hopkins University, Baltimore, MD

  2. 2

    Department of Neurology, Johns Hopkins University, Baltimore, MD

  3. 3

    Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institute of Health, National Institute of Health, Bethesda, MD

  4. 4

    Division of Veterinary Resources, Office of Research Services, National Institute of Health, Bethesda, MD

*Correspondence: Charlotte J. Sumner MD, Department of Neurology, Johns Hopkins University, Meyer 5-119b, 600 North Wolfe Street, Baltimore, MD 21287

Publication History

  1. Issue published online: 4 NOV 2008
  2. Article first published online: 25 JUL 2008
  3. Manuscript Accepted: 30 MAY 2008
  4. Manuscript Revised: 10 MAY 2008
  5. Manuscript Received: 26 FEB 2008

Funded by

  • National Institute of Neurological Disorders and Stroke (NINDS)
  • Career Transition Award. Grant Number: K22-NS0048199-01
  • NINDS Competitive Fellowship Award
  • NINDS Intramural Funds and Families of Spinal Muscular Atrophy, CJS

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Abstract

Early treatment with the histone deacetylase inhibitor, trichostatin A, plus nutritional support extended median survival of spinal muscular atrophy mice by 170%. Treated mice continued to gain weight, maintained stable motor function, and retained intact neuromuscular junctions long after trichostatin A was discontinued. In many cases, ultimate decline of mice appeared to result from vascular necrosis, raising the possibility that vascular dysfunction is part of the clinical spectrum of severe spinal muscular atrophy. Early spinal muscular atrophy disease detection and treatment initiation combined with aggressive ancillary care may be integral to the optimization of histone deacetylase inhibitor treatment in human patients. Ann Neurol 2008; 64:465–470

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