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Lu, D., Gong, X., Guo, X., Cai, Q., Chen, Y., Zhu, Y., et al. (2024) Gene Editing of the Endogenous Cryptic 3’ Splice Site Corrects the RNA Splicing Defect in the Β654-Thalassemia Mouse Model. Human Gene Therapy, 35, 825-837.
https://doi.org/10.1089/hum.2023.202

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