CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich's Ataxia.

Rocca, Celine J; Rainaldi, Joseph N; Sharma, Jay; Shi, Yanmeng; Haquang, Joseph H; Luebeck, Jens; Mali, Prashant; Cherqui, Stephanie

Rocca, Celine J; Rainaldi, Joseph N; Sharma, Jay; Shi, Yanmeng; Haquang, Joseph H; Luebeck, Jens; Mali, Prashant; Cherqui, Stephanie.

Afiliação

Rocca CJ; Division of Genetics, Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.
Rainaldi JN; Division of Genetics, Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.
Sharma J; Division of Genetics, Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.
Shi Y; Division of Genetics, Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.
Haquang JH; Division of Genetics, Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.
Luebeck J; Department of Bioengineering, University of California, San Diego, La Jolla, CA 92093, USA.
Mali P; Department of Bioengineering, University of California, San Diego, La Jolla, CA 92093, USA.
Cherqui S; Division of Genetics, Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.

Mol Ther Methods Clin Dev ; 17: 1026-1036, 2020 Jun 12.

Article em En | MEDLINE | ID: mdl-32462051

Palavras-chave

CRISPR/Cas9; Friedreich's ataxia; P53; ex vivo gene therapy; frataxin; gene editing; hematopoietic stem cells; mitochondrial disorder; neurodegenerative disorder

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Estados Unidos

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