A Deoxyribonucleic Acid Decoy Trapping DUX4 for the Treatment of Facioscapulohumeral Muscular Dystrophy

Facioscapulohumeral dystrophy (FSHD) is characterized by a loss of repressive epigenetic marks leading to the aberrant expression of the DUX4 transcription factor. In muscle, DUX4 acts as a poison protein though the induction of multiple downstream genes. So far, there is no therapeutic solution for...

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Detalles Bibliográficos
Autores principales: Mariot, Virginie, Joubert, Romain, Marsollier, Anne-Charlotte, Hourdé, Christophe, Voit, Thomas, Dumonceaux, Julie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7701011/
https://www.ncbi.nlm.nih.gov/pubmed/33312755
http://dx.doi.org/10.1016/j.omtn.2020.10.028

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