Validating UBC9 as a Molecular Target to Develop a Therapy for All Forms of ALS

Abstract

Our goal is to identify common mechanisms involved in neurodegeneration in ALS and to use that information to identify molecular targets to discover and develop a new class of therapeutics that block disease progression of all ALS patients. We identified a novel enzyme Ube2i (UBC9) that may be critical for mediating degeneration of motor neurons (MN) caused by different genes and proteins that are linked to both familial ALS (fALS) and sporadic ALS (sALS) pathogenesis. Since UBC9 is an enzyme, small molecule drugs designed to inhibit it could be effective in treating all forms of ALS. Our goal here is to evaluate UBC9 knockdown in cellular models of ALS for neuroprotective properties.

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Document Details

Document Type
Technical Report
Publication Date
Oct 01, 2019
Accession Number
AD1097377

Entities

People

  • Ashkan Javaherian
  • Steve Finkbeiner

Organizations

  • The J. David Gladstone Institutes

Tags

Communities of Interest

  • Autonomy
  • Energy and Power Technologies

DTIC Thesaurus Topics

  • Alzheimer Disease
  • Biological Sciences
  • Biomedical Research
  • Cell Physiological Processes
  • Cells
  • Dementia
  • Department Of Defense
  • Diseases And Disorders
  • Genetics
  • Hereditary Diseases
  • Information Systems
  • Medical Personnel
  • Neurodegeneration
  • Neurodegenerative Diseases
  • Parkinson'S Disease
  • Small Molecules
  • Therapy

Readers

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