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CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo
Huntington disease (HD) is a fatal dominantly inherited neurodegenerative disorder caused by CAG repeat expansion (>36 repeats) within the first exon of the huntingtin gene. Although mutant huntingtin (mHTT) is ubiquitously expressed, the brain shows robust and early degeneration. Current RNA int...
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| 出版年: | Mol Ther |
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| 主要な著者: | , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
American Society of Gene & Cell Therapy
2017
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5363210/ https://ncbi.nlm.nih.gov/pubmed/28129107 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2016.11.010 |
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