A research team from the Hubrecht Institute, Erasmus MC, and Sanquin has published the latest findings in Blood, developing a novel gene editing therapy called "delete-recruit." This technology reactivates dormant genes by adjusting the distance between genes and enhancers, providing new ideas for treating genetic blood diseases such as sickle cell disease and β-thalassemia.

The team utilized CRISPR-Cas9 gene editing to precisely cut specific DNA segments, bringing the dormant fetal globin gene closer to its enhancer. "We used scissors to cut out a segment of DNA between the enhancer and its gene, making the distance between them shorter," explained first author Anna-Karina Felder. Experiments confirmed that this method effectively reactivates fetal globin genes in blood stem cells from healthy donors and patients.

"Sickle cell disease or β-thalassemia patients have faulty adult globin genes. But fetal globin is like a backup engine," Felder stated. Reactivating this "backup engine" can promote healthy red blood cell production, potentially improving patient symptoms. Compared to existing therapies, this approach does not introduce exogenous genes, possibly offering better safety.

The technology's principle is simple yet innovative: achieving therapeutic effects solely by adjusting gene spacing, avoiding direct modification of gene sequences. The study shows significant effects in human cells, laying the foundation for safer, more cost-effective gene therapies. Beyond blood diseases, the technology could apply to other genetic conditions requiring activation of compensatory genes.