Article
Hypoimmunogenic human motor neurons induced from iPSCs in vivo substantially ameliorate ALS disease in large animal models
2025-09-07
Abstract excerpt
Stem cell-based therapy holds great potential for substituting degenerated motor neurons (MNs) in amyotrophic lateral sclerosis (ALS). Missing protocols for advanced differentiation of transplanted cells into MNs, immune rejection, and the lack of suitable ALS models for preclinical trials have slowed the development of effective therapies. Here, we employed multiplex genetic-editing to generate a novel human plur...
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Identifiers and source
- Literature Corpus work
- ee508119-2e41-5e77-b497-90e9e8e152c0
- DOI
- 10.1101/2025.09.03.673895
