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Chemical Modifications and Design Influence the Potency of Huntingtin Anti-Gene Oligonucleotides

Huntington's disease is a neurodegenerative, trinucleotide repeat (TNR) disorder affecting both males and females. It is caused by an abnormal increase in the length of CAG•CTG TNR in exon 1 of the Huntingtin gene (HTT). The resultant, mutant HTT mRNA and protein cause neuronal toxicity, sugges...

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Detalles Bibliográficos
Autores principales:	Saher, Osama, Zaghloul, Eman M., Umek, Tea, Hagey, Daniel W., Mozafari, Negin, Danielsen, Mathias B., Gouda, Alaa S., Lundin, Karin E., Jørgensen, Per T., Wengel, Jesper, Smith, C.I. Edvard, Zain, Rula
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2023
Materias:	Original Papers
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10066784/ https://www.ncbi.nlm.nih.gov/pubmed/36735581 http://dx.doi.org/10.1089/nat.2022.0046

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