Vertex Pharmaceuticals Incorporated
Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders

Abstract:

The present application provides materials and methods for treating a patient with one or more conditions associated with DMPK whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of DMPK gene in a cell by genome editing.