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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...

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Detalles Bibliográficos
Autores principales:	Shin, Jun Wan, Shin, Aram, Park, Seri S., Lee, Jong-Min
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
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

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