Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy

Facioscapulohumeral muscular dystrophy (FSHD), characterized by progressive muscle weakness and deterioration, is genetically linked to aberrant expression of DUX4 in muscle. DUX4, in its full-length form, is cytotoxic in nongermline tissues. Here, we designed locked nucleic acid (LNA) gapmer antise...

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Detalhes bibliográficos
Publicado no:Proc Natl Acad Sci U S A
Main Authors: Lim, Kenji Rowel Q., Maruyama, Rika, Echigoya, Yusuke, Nguyen, Quynh, Zhang, Aiping, Khawaja, Hunain, Sen Chandra, Sreetama, Jones, Takako, Jones, Peter, Chen, Yi-Wen, Yokota, Toshifumi
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2020
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7368245/
https://ncbi.nlm.nih.gov/pubmed/32601200
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1909649117
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