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Myotonic dystrophy type 1: from DNA repeat expansion and toxic RNA to the development of new therapeutic approaches

Archive ouverte | Gomes-Pereira, Mário | CCSD

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Cells of Matter—In Vitro Models for Myotonic Dystrophy

Archive ouverte | Matloka, Magdalena | CCSD

International audience. Myotonic dystrophy type 1 (DM1 also known as Steinert disease) is a multisystemic disorder mainly characterized by myotonia, progressive muscle weakness and wasting, cognitive impairments, an...

Switch-off the trouble:DMPK promoter targeting by CRISPRi as an original specific therapy in DM1

Archive ouverte | Porquet, Florent | CCSD

International audience. "Introduction: Myotonic dystrophy type 1 (DM1) originates from an amplification of CTG microsatellites in the DMPK gene. The pathology is primarily explained by a toxic gain of function where...

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