CRISPR correction of the Finnish ornithine delta-aminotransferase mutation restores metabolic homeostasis in iPSC from patients with gyrate atrophy.

Maldonado, Rocio; Jalil, Sami; Keskinen, Timo; Nieminen, Anni I; Hyvönen, Mervi E; Lapatto, Risto; Wartiovaara, Kirmo

Maldonado, Rocio; Jalil, Sami; Keskinen, Timo; Nieminen, Anni I; Hyvönen, Mervi E; Lapatto, Risto; Wartiovaara, Kirmo.

Afiliación

Maldonado R; Stem Cells and Metabolism Research Program, Faculty of Medicine, University of Helsinki, Helsinki, Finland.
Jalil S; Stem Cells and Metabolism Research Program, Faculty of Medicine, University of Helsinki, Helsinki, Finland.
Keskinen T; Stem Cells and Metabolism Research Program, Faculty of Medicine, University of Helsinki, Helsinki, Finland.
Nieminen AI; Metabolomics Unit, Institute for Molecular Medicine Finland, University of Helsinki, Helsinki, Finland.
Hyvönen ME; Stem Cells and Metabolism Research Program, Faculty of Medicine, University of Helsinki, Helsinki, Finland.
Lapatto R; Children's Hospital, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.
Wartiovaara K; Stem Cells and Metabolism Research Program, Faculty of Medicine, University of Helsinki, Helsinki, Finland.

Mol Genet Metab Rep ; 31: 100863, 2022 Jun.

Article en En | MEDLINE | ID: mdl-35782600

Palabras clave

CRISPR/Cas9; HOGA OAT; gene editing; gyrate atrophy

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Genet Metab Rep Año: 2022 Tipo del documento: Article País de afiliación: Finlandia Pais de publicación: Estados Unidos

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