Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

In recent years, hematopoietic stem cells gene editing has emerged as a promising tool to treat blood disorders. Here the authors develop a CRISPR/Cas9-based genome editing strategy that allows the precise correction of Wiskott-Aldrich Syndrome in vitro and in vivo with high efficiency.

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Auteurs principaux: Rajeev Rai, Marianna Romito, Elizabeth Rivers, Giandomenico Turchiano, Georges Blattner, Winston Vetharoy, Dariusz Ladon, Geoffroy Andrieux, Fang Zhang, Marta Zinicola, Diego Leon-Rico, Giorgia Santilli, Adrian J. Thrasher, Alessia Cavazza
Format: article
Langue:EN
Publié: Nature Portfolio 2020
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Science
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Accès en ligne:https://doaj.org/article/e93094c9c51f4c1aa746e611fd6ea64c
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Description
Résumé:In recent years, hematopoietic stem cells gene editing has emerged as a promising tool to treat blood disorders. Here the authors develop a CRISPR/Cas9-based genome editing strategy that allows the precise correction of Wiskott-Aldrich Syndrome in vitro and in vivo with high efficiency.

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