Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease.

Xu, Liwen; Lahiri, Premanjali; Skowronski, Jason; Bhatia, Neehar; Lattanzi, Annalisa; Porteus, Matthew H

Xu, Liwen; Lahiri, Premanjali; Skowronski, Jason; Bhatia, Neehar; Lattanzi, Annalisa; Porteus, Matthew H.

Afiliação

Xu L; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Lahiri P; Stanford Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
Skowronski J; Stanford Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
Bhatia N; Stanford Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
Lattanzi A; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Porteus MH; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.

Mol Ther Methods Clin Dev ; 30: 317-331, 2023 Sep 14.

Article em En | MEDLINE | ID: mdl-37637384

Palavras-chave

CRISPR; Cas9; HBB gene; SCD; genome editing; hematopoietic stem and progenitor cell; p53 activation; rAAV6

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Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Guideline Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos

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