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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Publicat a:Mol Ther Nucleic Acids
Autors principals: Provenzano, Claudia, Cappella, Marisa, Valaperta, Rea, Cardani, Rosanna, Meola, Giovanni, Martelli, Fabio, Cardinali, Beatrice, Falcone, Germana
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2017
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5684470/
https://ncbi.nlm.nih.gov/pubmed/29246312
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.10.006
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