Transplantation of Neural Progenitor Cells Expressing Glial Cell Line-Derived Neurotrophic Factor into the Motor Cortex as a Strategy to Treat Amyotrophic Lateral Sclerosis
Abstract
Early dysfunction of cortical motor neurons may underlie the initiation of amyotrophic lateral sclerosis (ALS). As such, the cortex represents a critical area of ALS research and a promising therapeutic target. In the current study, human cortical-derived neural progenitor cells engineered to secrete glial cell line-derived neurotrophic factor (GDNF) were transplanted into the SOD1G93A ALS rat cortex, where they migrated, matured into astrocytes, and released GDNF. This protected motor neurons, delayed disease pathology and extended survival of the animals. These same cells injected into the cortex of cynomolgus macaques survived and showed robust GDNF expression without adverse effects. Together this data suggests that introducing cortical astrocytes releasing GDNF represents a novel promising approach to treating ALS.
Document Details
- Document Type
- Pub Defense Publication
- Publication Date
- Apr 15, 2018
- Source ID
- 10.1002/stem.2825
Entities
People
- Annie A. Ma
- Clive N. Svendsen
- Gretchen M Thomsen
- Jean-philippe Vit
- Krystof S. Bankiewicz
- Livia Wyss
- Marlesa Godoy
- Mor Alkaslasi
- Noell Cho
- Oksana Shelest
- Pablo Avalos
- Patrick Suezaki
Organizations
- ALS Association
- Cedars-Sinai Medical Center
- United States Department of Defense
- University of California, San Francisco