Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.

Pickar-Oliver, Adrian; Gough, Veronica; Bohning, Joel D; Liu, Siyan; Robinson-Hamm, Jacqueline N; Daniels, Heather; Majoros, William H; Devlin, Garth; Asokan, Aravind; Gersbach, Charles A

Pickar-Oliver, Adrian; Gough, Veronica; Bohning, Joel D; Liu, Siyan; Robinson-Hamm, Jacqueline N; Daniels, Heather; Majoros, William H; Devlin, Garth; Asokan, Aravind; Gersbach, Charles A.

Afiliación

Pickar-Oliver A; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Gough V; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Bohning JD; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Liu S; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA; Graduate Program in Computational Biology and Bioinformatics, Duke University, Durham, NC 27708, USA.
Robinson-Hamm JN; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Daniels H; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Majoros WH; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA; Center for Statistical Genetics and Genomics, Duke University, Durham, NC 27708, USA; Division of Integrative Genomics, Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, NC
Devlin G; Department of Surgery, Duke University School of Medicine, Durham, NC 27710, USA.
Asokan A; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Department of Surgery, Duke University School of Medicine, Durham, NC 27710, USA; Regenerat
Gersbach CA; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA; Gradu

Mol Ther ; 29(11): 3243-3257, 2021 11 03.

Article en En | MEDLINE | ID: mdl-34509668

Asunto(s)

Sistemas CRISPR-Cas; Dependovirus/genética; Distrofina/genética; Exones; Edición Génica; Vectores Genéticos/genética; Distrofia Muscular de Duchenne/genética; Distrofia Muscular de Duchenne/terapia; Animales; Modelos Animales de Enfermedad; Expresión Génica; Orden Génico; Técnicas de Transferencia de Gen; Ingeniería Genética; Terapia Genética/métodos; Humanos; Ratones; Ratones Transgénicos; Músculo Esquelético/metabolismo; Mutación; Miocardio/metabolismo; Integración Viral

Palabras clave

CRISPR; genome editing; muscular dystrophy; targeted integration; viral vectors

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Exones / Distrofina / Dependovirus / Distrofia Muscular de Duchenne / Sistemas CRISPR-Cas / Edición Génica / Vectores Genéticos Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos

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