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Splicing biomarkers of disease severity in myotonic dystrophy.
Masayuki Nakamori
1 ,
Krzysztof Sobczak ,
Araya Puwanant ,
Steve Welle ,
Katy Eichinger ,
Shree Pandya ,
Jeannne Dekdebrun ,
Chad R Heatwole ,
Michael P McDermott ,
Tian Chen ,
Melissa Cline ,
Rabi Tawil ,
Robert J Osborne ,
Thurman M Wheeler ,
Maurice S Swanson ,
Richard T Moxley ,
Charles A Thornton
Dec 2013
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Abstract
To develop RNA splicing biomarkers of disease severity and therapeutic response in myotonic dystrophy type 1 (DM1) and type 2 (DM2).