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Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9
A comprehensive genetics-based precision medicine strategy to selectively and permanently inactivate only mutant, not normal allele, could benefit many dominantly inherited disorders. Here, we demonstrate the power of our novel strategy of inactivating the mutant allele using haplotype-specific CRIS...
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| Publicado no: | Hum Mol Genet |
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| Main Authors: | , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Oxford University Press
2016
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6078600/ https://ncbi.nlm.nih.gov/pubmed/28172889 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddw286 |
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