Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges.

Miccio, Annarita; Antoniou, Panagiotis; Ciura, Sorana; Kabashi, Edor

Miccio, Annarita; Antoniou, Panagiotis; Ciura, Sorana; Kabashi, Edor.

Afiliação

Miccio A; Laboratory of Chromatin and Gene Regulation during Development, Imagine Institute, Université de Paris, INSERM UMR 1163, 75015, Paris, France. Electronic address: annarita.miccio@institutimagine.org.
Antoniou P; Laboratory of Chromatin and Gene Regulation during Development, Imagine Institute, Université de Paris, INSERM UMR 1163, 75015, Paris, France.
Ciura S; Laboratory of Translational Research for Neurological Disorders, Imagine Institute, Université de Paris, INSERM UMR 1163, 75015, Paris, France.
Kabashi E; Laboratory of Translational Research for Neurological Disorders, Imagine Institute, Université de Paris, INSERM UMR 1163, 75015, Paris, France. Electronic address: edor.kabashi@institutimagine.org.

Mol Ther ; 30(1): 47-53, 2022 01 05.

Article em En | MEDLINE | ID: mdl-33823304

Assuntos

Esclerose Lateral Amiotrófica; Atrofia Muscular Espinal; Esclerose Lateral Amiotrófica/genética; Esclerose Lateral Amiotrófica/metabolismo; Esclerose Lateral Amiotrófica/terapia; Edição de Genes; Humanos; Lactente; Atrofia Muscular Espinal/genética; Atrofia Muscular Espinal/metabolismo; Atrofia Muscular Espinal/terapia; Oligonucleotídeos/metabolismo; Splicing de RNA; Proteína 1 de Sobrevivência do Neurônio Motor/genética; Proteína 1 de Sobrevivência do Neurônio Motor/metabolismo

Palavras-chave

ALS; CRISPR; SMA; base editing; genome editing; motor neuron diseases

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Esclerose Lateral Amiotrófica Limite: Humans / Infant Idioma: En Ano de publicação: 2022 Tipo de documento: Article

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