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Lentivirus-mediated gene therapy corrects ribosomal biogenesis and shows promise for Diamond Blackfan anemia.

JCI Insight · 2024 · PMC11141922 · PMID 38775150

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In these cultures, a higher output of mature erythropoietic progenitors (CD71 – CD2352a + ) was observed, which, however, did not reach statistical significance ( Figure 4, F and G ).

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