Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models.

Mol Ther Methods Clin Dev · 2025 · PMC11964741 · PMID 40176947

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highly significantno p-value reported

Overall, we observed a highly significant decrease in LMO2 expression in both GbG M and GbG MI group when compared to the SFFV RV group ( Figure 4 A).

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