Utrophin up-regulation by artificial transcription factors induces muscle rescue and impacts the neuromuscular junction in mdx mice.

Pisani, Cinzia; Strimpakos, Georgios; Gabanella, Francesca; Di Certo, Maria Grazia; Onori, Annalisa; Severini, Cinzia; Luvisetto, Siro; Farioli-Vecchioli, Stefano; Carrozzo, Irene; Esposito, Antonio; Canu, Tamara; Mattei, Elisabetta; Corbi, Nicoletta; Passananti, Claudio

Pisani, Cinzia; Strimpakos, Georgios; Gabanella, Francesca; Di Certo, Maria Grazia; Onori, Annalisa; Severini, Cinzia; Luvisetto, Siro; Farioli-Vecchioli, Stefano; Carrozzo, Irene; Esposito, Antonio; Canu, Tamara; Mattei, Elisabetta; Corbi, Nicoletta; Passananti, Claudio.

Afiliação

Pisani C; CNR-Institute of Molecular Biology and Pathology, Department of Molecular Medicine, Sapienza University, Rome, Italy. Electronic address: cinzia.pisani@uniroma1.it.
Strimpakos G; CNR-Cell Biology and Neurobiology Institute, Rome, Italy; IRCCS Santa Lucia Foundation, Rome, Italy.
Gabanella F; CNR-Cell Biology and Neurobiology Institute, Rome, Italy.
Di Certo MG; CNR-Cell Biology and Neurobiology Institute, Rome, Italy.
Onori A; CNR-Institute of Molecular Biology and Pathology, Department of Molecular Medicine, Sapienza University, Rome, Italy.
Severini C; CNR-Cell Biology and Neurobiology Institute, Rome, Italy.
Luvisetto S; CNR-Cell Biology and Neurobiology Institute, Rome, Italy; IRCCS Santa Lucia Foundation, Rome, Italy.
Farioli-Vecchioli S; CNR-Cell Biology and Neurobiology Institute, Rome, Italy; IRCCS Santa Lucia Foundation, Rome, Italy.
Carrozzo I; CNR-Institute of Molecular Biology and Pathology, Department of Molecular Medicine, Sapienza University, Rome, Italy.
Esposito A; Preclinical Imaging Facility, Experimental Imaging Center, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Italy.
Canu T; Preclinical Imaging Facility, Experimental Imaging Center, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Italy.
Mattei E; CNR-Cell Biology and Neurobiology Institute, Rome, Italy; IRCCS Santa Lucia Foundation, Rome, Italy.
Corbi N; CNR-Institute of Molecular Biology and Pathology, Department of Molecular Medicine, Sapienza University, Rome, Italy.
Passananti C; CNR-Institute of Molecular Biology and Pathology, Department of Molecular Medicine, Sapienza University, Rome, Italy. Electronic address: claudio.passananti@uniroma1.it.

Biochim Biophys Acta Mol Basis Dis ; 1864(4 Pt A): 1172-1182, 2018 Apr.

Article em En | MEDLINE | ID: mdl-29408646

Assuntos

Terapia Genética/métodos; Músculo Esquelético/metabolismo; Distrofia Muscular de Duchenne/terapia; Junção Neuromuscular/metabolismo; Engenharia de Proteínas; Fatores de Transcrição; Regulação para Cima; Animais; Células HeLa; Humanos; Camundongos; Camundongos Endogâmicos mdx; Músculo Esquelético/patologia; Distrofia Muscular de Duchenne/genética; Distrofia Muscular de Duchenne/metabolismo; Distrofia Muscular de Duchenne/patologia; Junção Neuromuscular/genética; Junção Neuromuscular/patologia; Fatores de Transcrição/biossíntese; Fatores de Transcrição/genética; Utrofina/genética; Dedos de Zinco

Palavras-chave

AAV; DMD; Gene therapy; NMJ; Utrophin; ZF-ATF

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Fatores de Transcrição / Terapia Genética / Engenharia de Proteínas / Regulação para Cima / Músculo Esquelético / Distrofia Muscular de Duchenne / Junção Neuromuscular Limite: Animals / Humans Idioma: En Revista: Biochim Biophys Acta Mol Basis Dis Ano de publicação: 2018 Tipo de documento: Article

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