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 gene....

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Bibliographic Details
Main Authors: Claudia Provenzano, Marisa Cappella, Rea Valaperta, Rosanna Cardani, Giovanni Meola, Fabio Martelli, Beatrice Cardinali, Germana Falcone
Format: Article
Language:English
Published: Elsevier 2017-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
myotonic dystrophy type 1
CRISPR/Cas9
trinucleotide expansion
splicing alterations
phenotypic reversion
DM1 cell models
DMPK
CTG repeats
muscle disease
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117302676

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http://www.sciencedirect.com/science/article/pii/S2162253117302676

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