Vertex Pharmaceuticals Incorporated
CRISPR/CAS ALL-IN-TWO VECTOR SYSTEMS FOR TREATMENT OF DMD

Abstract:

The present disclosure provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD), e.g., through ex vivo and in vivo methods of genome editing. The present disclosure also relates to methods and compositions for use of self-inactivating/self-targeting CRISPR/Cas or CRISPR/Cpf1 systems to genetically modify cells, e.g., to modulate the expression, function, and/or activity of the dystrophin gene.