In the not-yet-peer-reviewed study, the <a href="https://healthylife7.com/on-her-mind-medical-research-catching-up-to-womens-health/” title=”On Her Mind: Medical research catching up to women's health”>researchers targeted the erythroid-specific +58 kb enhancer of BCL11A, whose disruption relieves repression of γ-globin and raises fetal haemoglobin. They treated primary CD34+ HSPCs from three healthy HbAA donors and two donors with sickle cell disease using either Cas9-sgRNA ribonucleoprotein electroporation or lipid nanoparticles carrying Cas9 mRNA and the same sgRNA
Targeted sequencing showed up to 74% modified alleles after electroporation versus 41% after LNP delivery; during differentiation, rates peaked at 90% and 56%, respectively, in one healthy-donor cohort. However, LNP-treated cells recovered better after editing and yielded more mature red cells
In HbSS-derived cells, LNP editing generated about 14% HbF and reduced sickling under hypoxia despite little change in bulk oxygen affinity. Enucleation reached 99.2% after LNP editing versus 97.2% after electroporation, while mature red-cell recovery after filtration exceeded 6% for LNP-treated cells but was 0.6% after electroporation
The study was led by Yaw Ansong-Ansongton and David Nguyen at the University of California, Berkeley, and the University of California, San Francisco. It was published in bioRxiv on 31 August 2026
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ArticleCMN BriefsNewsCMN BriefsNon-viralElectroporationLipid-based nanoparticleSickle Cell Disease, SCDBlood diseaseCas9
