Gene Editing/Gene Therapies: NON-VIRAL DNA DELIVERY ASSOCIATED TO TALEN® GENE EDITING LEADS TO HIGHLY EFFICIENT CORRECTION OF SICKLE CELL MUTATION IN LONG-TERM REPOPULATING HEMATOPOIETIC STEM CELLS

Arianna Moiani, Gaëlle Letort,Sabrina Lizot, Patrick Hong, S. Temburni-Black, Tainara F. Felix,Anne Chalumeau,Annarita Miccio, A. Gouble,Alexandre Juillerat, Aymeric Duclert,Philippe Duchâteau,Julien Valton

Cytotherapy(2023)

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关键词
sickle cell mutation,gene therapies,stem cells,non-viral,long-term
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