Nuclear accumulation of CHMP7 initiates nuclear pore complex injury and subsequent TDP-43 dysfunction in sporadic and familial ALS
Abstract
Nuclear accumulation of CHMP7 leads to multiple cellular pathologies that can be mitigated via CHMP7 ASOs in familial and sporadic ALS iPSNs.
Document Details
- Document Type
- Pub Defense Publication
- Publication Date
- Jul 28, 2021
- Source ID
- 10.1126/scitranslmed.abe1923
Entities
People
- Alyssa N. Coyne
- Benjamin L. Zaepfel
- C Frank Bennett
- C Patrick Lusk
- Dennis W. Dickson
- Frank Rigo
- Jeffrey D Rothstein
- Victoria Baskerville
Organizations
- ALS Association
- Chan Zuckerberg Initiative
- Fidelity Foundation
- Ionis Pharmaceuticals
- Johns Hopkins University
- Mayo Clinic
- Muscular Dystrophy Association
- National Institutes of Health
- United States Department of Defense
- Yale School of Medicine