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August 31, 2024Molecular Therapy — Nucleic AcidsOpen Access

Cas9 Editing of ATXN1 in a Spinocerebellar Ataxia type 1 Mice and human iPSC-derived neurons

K Fagan Guillem Chillon Ellie M. Carrell Elisa A. Waxman Beverly L. Davidson

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Abstract

Spinocerebellar ataxia type 1 (SCA1) is an adult-onset neurodegenerative disease caused by an expansion of the CAG repeat region of the

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Cite This Study

Fagan et al. (2024) studied this question.

synapsesocial.com/papers/68e5a0adb6db64358753b784 https://doi.org/10.1016/j.omtn.2024.102317

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Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Disruption of the ATXN1-CIC complex reveals the role of additional nuclear ATXN1 interactors in spinocerebellar ataxia type 1Original Research · 2022 · 28 citations
2Translational Relevance of SCA1 Models for the Development of Therapies for Spinocerebellar Ataxia Type 12025 · 1 citations
3Gene, Stem Cell, and Alternative Therapies for SCA 1Review · 2016 · 14 citations
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