Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies.
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Venkatesan V, et al. Among authors: nakamura y.
Mol Ther Nucleic Acids. 2023 Apr 26;32:671-688. doi: 10.1016/j.omtn.2023.04.024. eCollection 2023 Jun 13.
Mol Ther Nucleic Acids. 2023.
PMID: 37215154
Free PMC article.