Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X-Linked (mdx) Mouse.

Zarrouki, Faouzi; Relizani, Karima; Bizot, Flavien; Tensorer, Thomas; Garcia, Luis; Vaillend, Cyrille; Goyenvalle, Aurélie

Zarrouki, Faouzi; Relizani, Karima; Bizot, Flavien; Tensorer, Thomas; Garcia, Luis; Vaillend, Cyrille; Goyenvalle, Aurélie.

Afiliação

Zarrouki F; UVSQ, Inserm, END-ICAP, Université Paris-Saclay, Versailles, France.
Relizani K; CNRS, Institut des Neurosciences Paris Saclay, Université Paris-Saclay, Saclay, France.
Bizot F; UVSQ, Inserm, END-ICAP, Université Paris-Saclay, Versailles, France.
Tensorer T; SQY Therapeutics, UVSQ, Montigny le Bretonneux, France.
Garcia L; UVSQ, Inserm, END-ICAP, Université Paris-Saclay, Versailles, France.
Vaillend C; SQY Therapeutics, UVSQ, Montigny le Bretonneux, France.
Goyenvalle A; UVSQ, Inserm, END-ICAP, Université Paris-Saclay, Versailles, France.

Ann Neurol ; 92(2): 213-229, 2022 08.

Article em En | MEDLINE | ID: mdl-35587226

Assuntos

Distrofina; Distrofia Muscular de Duchenne; Animais; Encéfalo/metabolismo; Modelos Animais de Doenças; Distrofina/genética; Distrofina/metabolismo; Éxons/genética; Camundongos; Camundongos Endogâmicos mdx; Distrofia Muscular de Duchenne/genética; Distrofia Muscular de Duchenne/terapia; Oligonucleotídeos; Oligonucleotídeos Antissenso; Distribuição Tecidual

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofina / Distrofia Muscular de Duchenne Limite: Animals Idioma: En Ano de publicação: 2022 Tipo de documento: Article

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