Article
An isogenetic myoblast expression screen identifies DUX4-mediated FSHD-associated molecular pathologies.
The EMBO journal - 22 Oct 2008
Bosnakovski Darko, Xu Zhaohui, Gang Eun Ji, Galindo Cristi L, Liu Mingju, Simsek Tugba, Garner Harold R, Agha-Mohammadi Siamak, Tassin Alexandra, Coppée Frédérique, Belayew Alexandra, Perlingeiro Rita R, Kyba Michael
Abstract excerpt
Facioscapulohumeral muscular dystrophy (FSHD) is caused by an unusual deletion with neomorphic activity. This deletion derepresses genes in cis; however which candidate gene causes the FSHD phenotype, and through what mechanism, is unknown. We describe a novel genetic tool, inducible cassette exchange, enabling rapid generation of isogenetically modified cells with conditional and variable transgene expression....
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