RNA-Targeting CRISPR/CasRx system relieves disease symptoms in Huntington’s disease models
Abstract Background HD is a devastating neurodegenerative disorder caused by the expansion of CAG repeats in the HTT. Silencing the expression of mutated proteins is a therapeutic direction to rescue HD patients, and recent advances in gene editing technology such as CRISPR/CasRx have opened up new...
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| Hauptverfasser: | , , , , , , , , , , , , , , , , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
BMC
2025-01-01
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| Schriftenreihe: | Molecular Neurodegeneration |
| Schlagworte: | |
| Online-Zugang: | https://doi.org/10.1186/s13024-024-00794-w |
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