Journal article
Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds
Abstract
Myotonic dystrophy type 1 (DM1) and type 2 (DM2) are autosomal dominant neuromuscular diseases caused by microsatellite expansions and belong to the family of RNA-dominant disorders. Availability of cellular models in which the DM mutation is expressed within its natural context is essential to facilitate efforts to identify new therapeutic compounds. Here, we generated immortalized DM1 and DM2 human muscle cell lines that display nuclear RNA …
Authors
Arandel L; Espinoza MP; Matloka M; Bazinet A; De Dea Diniz D; Naouar N; Rau F; Jollet A; Edom-Vovard F; Mamchaoui K
Journal
Disease Models & Mechanisms, Vol. 10, No. 4, pp. 487–497
Publisher
The Company of Biologists
Publication Date
April 1, 2017
DOI
10.1242/dmm.027367
ISSN
1754-8403