Cas9 editing of ATXN1 in a spinocerebellar ataxia type 1 mice and human iPSC-derived neurons
Spinocerebellar ataxia type 1 (SCA1) is an adult-onset neurodegenerative disease caused by an expansion of the CAG repeat region of the ATXN1 gene. Currently there are no disease-modifying treatments; however, previous work has shown the potential of gene therapy, specifically RNAi, as a potential m...
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| Main Authors: | , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2024-12-01T00:00:00Z.
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| Online Access: | Connect to this object online. |
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