Article

Clinical improvement of DM1 patients reflected by reversal of disease-induced gene expression in blood

2022-03-12

Abstract excerpt

Myotonic dystrophy type 1 (DM1) is an incurable multisystem disease caused by a CTG-repeat expansion in the DM1 protein kinase ( DMPK ) gene. The OPTIMISTIC clinical trial demonstrated positive and heterogenous effects of cognitive behavioral therapy (CBT) on the capacity for activity and social participations in DM1 patients. Here, we performed mRNA sequencing of full blood for 27 patients of the OPTIMISTIC cohor...

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Literature Corpus work: 6ebfc440-6f86-597a-bd43-e903d1543af2
DOI: 10.1101/2022.03.11.22272021

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