Contracting CAG/CTG repeats using the CRISPR-Cas9 nickase
Abstract CAG/CTG repeat expansions cause over 13 neurological diseases that remain without a cure. Because longer tracts cause more severe phenotypes, contracting them may provide a therapeutic avenue. No currently known agent can specifically generate contractions. Using a GFP-based chromosomal rep...
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| Autori principali: | , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Nature Portfolio
2016-11-01
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| Serie: | Nature Communications |
| Accesso online: | https://doi.org/10.1038/ncomms13272 |
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