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Transplantation of Neural Progenitor Cells Expressing Glial Cell Line-Derived Neurotrophic Factor Into the Motor Cortex as a Strategy to Treat Amyotrophic Lateral Sclerosis

Gretchen M Thomsen, Pablo Avalos, Annie A Ma, Mor Alkaslasi, Noell Cho, Livia Wyss, Jean-Philippe Vit, Marlesa Godoy, Patrick Suezaki, Oksana Shelest, Krystof S Bankiewicz, Clive N Svendsen

Stem Cells. 2018 Jul;36(7):1122-1131.

PMID: 29656478

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. Stem Cells 2018;36:1122-1131.

Chemicals Related in the Paper:

Catalog Number Product Name Structure CAS Number Price
IAR4248902 Glial Cell Line-derived Neurotrophic Factor from rat Glial Cell Line-derived Neurotrophic Factor from rat Price
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