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CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients.
Provenzano, Claudia; Cappella, Marisa; Valaperta, Rea; Cardani, Rosanna; Meola, Giovanni; Martelli, Fabio; Cardinali, Beatrice; Falcone, Germana.
Affiliation
  • Provenzano C; Institute of Cell Biology and Neurobiology, National Research Council, Monterotondo, Rome, Italy.
  • Cappella M; Institute of Cell Biology and Neurobiology, National Research Council, Monterotondo, Rome, Italy; DAHFMO-Unit of Histology and Medical Embryology, Sapienza University of Rome, Rome, Italy.
  • Valaperta R; Molecular Biology Laboratory, Policlinico San Donato-IRCCS, San Donato Milanese, Milan, Italy.
  • Cardani R; Muscle Histopathology and Molecular Biology Laboratory, Policlinico San Donato-IRCCS, San Donato Milanese, Milan, Italy.
  • Meola G; Department of Neurology, IRCCS Policlinico San Donato, San Donato Milanese, Milan, Italy; Department of Biomedical Sciences for Health, University of Milan, Milan, Italy.
  • Martelli F; Molecular Cardiology Laboratory, Policlinico San Donato-IRCCS, San Donato Milanese, Milan, Italy.
  • Cardinali B; Institute of Cell Biology and Neurobiology, National Research Council, Monterotondo, Rome, Italy. Electronic address: beatrice.cardinali@cnr.it.
  • Falcone G; Institute of Cell Biology and Neurobiology, National Research Council, Monterotondo, Rome, Italy. Electronic address: germana.falcone@cnr.it.
Mol Ther Nucleic Acids ; 9: 337-348, 2017 Dec 15.
Article in En | MEDLINE | ID: mdl-29246312
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2017 Document type: Article Affiliation country: Italy Country of publication: United States
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2017 Document type: Article Affiliation country: Italy Country of publication: United States