Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS

Autosomal dominant diseases such as Huntington’s disease (HD) are caused by a gain of function mutant protein and/or RNA. An ideal treatment for these diseases is to selectively suppress expression of the mutant allele while preserving expression of the wild-type variant. RNase H active antisense ol...

Celý popis

Uloženo v:
Podrobná bibliografie
Hlavní autoři: Østergaard, Michael E., Southwell, Amber L., Kordasiewicz, Holly, Watt, Andrew T., Skotte, Niels H., Doty, Crystal N., Vaid, Kuljeet, Villanueva, Erika B., Swayze, Eric E., Frank Bennett, C., Hayden, Michael R., Seth, Punit P.
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2013
Témata:
Gene Regulation, Chromatin and Epigenetics
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3834808/
https://ncbi.nlm.nih.gov/pubmed/23963702
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkt725
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky