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....

সম্পূর্ণ বিবরণ

গ্রন্থ-পঞ্জীর বিবরন
প্রধান লেখক: Claudia Provenzano, Marisa Cappella, Rea Valaperta, Rosanna Cardani, Giovanni Meola, Fabio Martelli, Beatrice Cardinali, Germana Falcone
বিন্যাস: প্রবন্ধ
ভাষা:English
প্রকাশিত: Elsevier 2017-12-01
মালা:Molecular Therapy: Nucleic Acids
বিষয়গুলি:
myotonic dystrophy type 1
CRISPR/Cas9
trinucleotide expansion
splicing alterations
phenotypic reversion
DM1 cell models
DMPK
CTG repeats
muscle disease
অনলাইন ব্যবহার করুন:http://www.sciencedirect.com/science/article/pii/S2162253117302676

আন্তর্জাল

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

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