Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
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Mol Ther Methods Clin Dev. 2021 May 14;22:237-248. doi: 10.1016/j.omtm.2021.05.001. eCollection 2021 Sep 10.
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PMID: 34485608
Free PMC article.