Transiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications.

Pini, Veronica; Mariot, Virginie; Dumonceaux, Julie; Counsell, John; O'Neill, Helen C; Farmer, Sarah; Conti, Francesco; Muntoni, Francesco

Pini, Veronica; Mariot, Virginie; Dumonceaux, Julie; Counsell, John; O'Neill, Helen C; Farmer, Sarah; Conti, Francesco; Muntoni, Francesco.

Afiliação

Pini V; Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, WC1N 1EH, UK. v.pini@ucl.ac.uk.
Mariot V; Translational Myology Laboratory, Molecular Neurosciences Section, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, WC1N 1EH, UK.
Dumonceaux J; Translational Myology Laboratory, Molecular Neurosciences Section, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, WC1N 1EH, UK.
Counsell J; Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, WC1N 1EH, UK.
O'Neill HC; Genome Editing and Reproductive Genetics Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WC1E 6HX, UK.
Farmer S; Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, WC1N 1EH, UK.
Conti F; Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, WC1N 1EH, UK.
Muntoni F; Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, WC1N 1EH, UK. f.muntoni@ucl.ac.uk.

Sci Rep ; 12(1): 3756, 2022 03 08.

Article em En | MEDLINE | ID: mdl-35260651

Assuntos

Sistemas CRISPR-Cas; Distrofia Muscular de Duchenne; Sistemas CRISPR-Cas/genética; Distrofina/genética; Distrofina/metabolismo; Endonucleases/genética; Edição de Genes/métodos; Terapia Genética/métodos; Humanos; Distrofia Muscular de Duchenne/genética; Distrofia Muscular de Duchenne/metabolismo; Distrofia Muscular de Duchenne/terapia; Mioblastos/metabolismo

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne / Sistemas CRISPR-Cas Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article

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