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Targeting N-Terminal Huntingtin with a Dual-sgRNA Strategy by CRISPR/Cas9
Huntington's disease (HD) is an autosomal dominant progressive neurodegenerative disorder, caused by a CAG/polyglutamine (polyQ) repeat expansion in the Huntingtin (HTT) gene. The polyQ tract is located in and transcribed from N-terminal HTT of exon 1. HTT is a large multifaceted protein, which...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Hindawi
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6881766/ https://www.ncbi.nlm.nih.gov/pubmed/31828084 http://dx.doi.org/10.1155/2019/1039623 |