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Time-controlled and muscle-specific CRISPR/Cas9-mediated deletion of CTG-repeat expansion in the DMPK gene.
Cardinali, Beatrice; Provenzano, Claudia; Izzo, Mariapaola; Voellenkle, Christine; Battistini, Jonathan; Strimpakos, Georgios; Golini, Elisabetta; Mandillo, Silvia; Scavizzi, Ferdinando; Raspa, Marcello; Perfetti, Alessandra; Baci, Denisa; Lazarevic, Dejan; Garcia-Manteiga, Jose Manuel; Gourdon, Geneviève; Martelli, Fabio; Falcone, Germana.
Afiliação
  • Cardinali B; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
  • Provenzano C; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
  • Izzo M; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
  • Voellenkle C; Molecular Cardiology Laboratory, IRCCS Policlinico San Donato, San Donato Milanese, 20097 Milan, Italy.
  • Battistini J; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
  • Strimpakos G; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
  • Golini E; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
  • Mandillo S; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
  • Scavizzi F; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
  • Raspa M; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
  • Perfetti A; Molecular Cardiology Laboratory, IRCCS Policlinico San Donato, San Donato Milanese, 20097 Milan, Italy.
  • Baci D; Molecular Cardiology Laboratory, IRCCS Policlinico San Donato, San Donato Milanese, 20097 Milan, Italy.
  • Lazarevic D; Center for Omics Sciences, IRCCS Ospedale San Raffaele, 20132 Milan, Italy.
  • Garcia-Manteiga JM; Center for Omics Sciences, IRCCS Ospedale San Raffaele, 20132 Milan, Italy.
  • Gourdon G; Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, 75013 Paris, France.
  • Martelli F; Molecular Cardiology Laboratory, IRCCS Policlinico San Donato, San Donato Milanese, 20097 Milan, Italy.
  • Falcone G; Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy.
Mol Ther Nucleic Acids ; 27: 184-199, 2022 Mar 08.
Article em En | MEDLINE | ID: mdl-34976437

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Itália País de publicação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Itália País de publicação: Estados Unidos