CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients

CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients

Myotonic dystrophy type 1 (DM1) is the most common adult-onset muscular dystrophy, characterized by progressive myopathy, myotonia, and multi-organ involvement. This dystrophy is an inherited autosomal dominant disease caused by a (CTG)n expansion within the 3' untranslated region of the DMPK g...

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Main Authors: Claudia Provenzano (Author), Marisa Cappella (Author), Rea Valaperta (Author), Rosanna Cardani (Author), Giovanni Meola (Author), Fabio Martelli (Author), Beatrice Cardinali (Author), Germana Falcone (Author)
Format: Book
Published: Elsevier, 2017-12-01T00:00:00Z.
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