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Haplotype-specific insertion-deletion variations for allele-specific targeting in Huntington's disease
Huntington's disease (HD) is a dominantly inherited neurodegenerative disease caused by an expanded CAG repeat in huntingtin (HTT). Given an important role for HTT in development and significant neurodegeneration at the time of clinical manifestation in HD, early treatment of allele-specific dr...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8933729/ https://www.ncbi.nlm.nih.gov/pubmed/35356757 http://dx.doi.org/10.1016/j.omtm.2022.03.001 |