Developing Wide-Spectrum Antiproteotoxicity Agents to Treat ALS

Abstract

ALS remains a devastating neurodegenerative disease with no curative treatments available. It is becoming increasingly clear that the accumulation of misfolded and aggregated proteins underlie the pathologies of several major forms of ALS. Protein products of ALS genes, including SOD1 and TDP-43, have been involved in the pathology of protein aggregation. In the past year of research, we have made significant progress towards identifying novel therapeutic agents against protein aggregation and delivering the agents to the relevant nervous systems. Our work has revealed that inhibitors of specific targets have robust effects on protein aggregation in a protein-dependent manner. We have also demonstrated significant progress in delivering agents to in vivo animal models. Further studies are required to pursue some of the new findings that were observed.

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Document Details

Document Type
Technical Report
Publication Date
Oct 01, 2013
Accession Number
ADA601368

Entities

People

  • Goran Periz
  • Jiou Wang
  • John M. Shea
  • Qingwen Kawaji
  • Sarah Kavianpour
  • Zhang Tao

Organizations

  • Johns Hopkins University

Tags

DTIC Thesaurus Topics

  • Cells
  • Central Nervous System
  • Chemical Synthesis
  • Chemistry
  • Control Systems
  • Diseases And Disorders
  • Gene Expression
  • Gene Therapy
  • Genetics
  • Inhibitors
  • Intranuclear Space
  • Metabolic Diseases
  • Nervous System
  • Neurodegenerative Diseases
  • Proteins
  • Small Molecules
  • Spinal Cord

Fields of Study

  • Biology

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