In this week's episode,
Blood editor Dr. James Griffin interviews Drs. George Goshua, Gerd Blobel, and Paul Kaminski on their latest articles published in
Blood. Dr. Goshua elaborates on the background and then insights from "
Haploidentical transplant, gene therapy, and standard care in sickle cell disease: a cost-effectiveness analysis". This analysis provides valuable guidance for clinicians, patients, and health systems as they consider treatment choices. However, as concluded in the accompanying
Blood Commentary, the true measure of success is not which therapy “wins” the economic argument, but whether each patient receives the therapy best suited to their clinical needs and values. Then, Drs. Gerd Blobel and Paul Kaminski share "
Dissecting polycomb complexes for enhanced fetal hemoglobin production", which utilizes a comprehensive CRISPR-based screen to interrogate the components of these repressive complexes and identified a single protein domain in EZH2, a subunit of PRC2, as a potential therapeutic target. They demonstrate that inhibition of the domain encoded by exon 14 of EZH2 selectively derepresses fetal hemoglobin expression, raising the possibility of developing drugs that specifically target this domain to treat hemoglobinopathies.