A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.

Biferi, Maria Grazia; Cohen-Tannoudji, Mathilde; Cappelletto, Ambra; Giroux, Benoit; Roda, Marianne; Astord, Stéphanie; Marais, Thibaut; Bos, Corinne; Voit, Thomas; Ferry, Arnaud; Barkats, Martine

Biferi, Maria Grazia; Cohen-Tannoudji, Mathilde; Cappelletto, Ambra; Giroux, Benoit; Roda, Marianne; Astord, Stéphanie; Marais, Thibaut; Bos, Corinne; Voit, Thomas; Ferry, Arnaud; Barkats, Martine.

Afiliação

Biferi MG; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France. Electronic address: mg.biferi@institut-myologie.org.
Cohen-Tannoudji M; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
Cappelletto A; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
Giroux B; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
Roda M; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
Astord S; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
Marais T; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
Bos C; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
Voit T; NIHR GOSH Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, London WC1N 1EH, UK.
Ferry A; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France; Sorbonne Paris Cité, Université Paris Descartes, Paris 75006, France.
Barkats M; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.

Mol Ther ; 25(9): 2038-2052, 2017 09 06.

Article em En | MEDLINE | ID: mdl-28663100

Assuntos

Esclerose Lateral Amiotrófica/genética; Dependovirus/genética; Terapia Genética; Vetores Genéticos/genética; Superóxido Dismutase-1/genética; Idade de Início; Esclerose Lateral Amiotrófica/mortalidade; Esclerose Lateral Amiotrófica/fisiopatologia; Esclerose Lateral Amiotrófica/terapia; Animais; Modelos Animais de Doenças; Éxons; Ordem dos Genes; Técnicas de Transferência de Genes; Terapia Genética/métodos; Vetores Genéticos/administração & dosagem; Humanos; Camundongos; Camundongos Transgênicos; Atividade Motora/genética; Oligonucleotídeos Antissenso; Sítios de Splice de RNA; RNA Mensageiro/genética; RNA Mensageiro/metabolismo; Recuperação de Função Fisiológica; Superóxido Dismutase-1/metabolismo; Taxa de Sobrevida; Transdução Genética

Palavras-chave

AAV; ALS; exon skipping; gene therapy; hSOD1

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Superóxido Dismutase-1 / Vetores Genéticos / Esclerose Lateral Amiotrófica Idioma: En Ano de publicação: 2017 Tipo de documento: Article

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