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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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Literature Corpus work
ee508119-2e41-5e77-b497-90e9e8e152c0
DOI
10.1101/2025.09.03.673895
Open publication

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Hypoimmunogenic human motor neurons induced from iPSCs in vivo substantially ameliorate ALS disease in large animal modelsDOI 10.1101/2025.09.03.673895
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