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Two is better than one: Advancing gene therapy protocols for enhanced safety and efficacy
Giulia Hardouin1, Annarita Miccio1
1Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR1163, Paris, France.
Cell Stem Cell
|February 7, 2025
Abstract:
In this issue of Cell Stem Cell, two complementary studies from Zeng et al.1 and Demirci et al.2 improve the efficacy of gene therapy for β-hemoglobinopathies using multiplex CRISPR-Cas9 editing. The authors also optimize editing and conditioning protocols to reduce risks associated with current gene therapy procedures.
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