Gene therapy targets sickle-cell disease – Nature

Image credit: Steve Babuljak/UCSF

Gene therapy might offer a cure for sickle-cell disease, and clinical trials are already under way. The approach is promising because just a single gene needs correcting: the one for the β-globin subunit of haemoglobin, the body’s oxygen ferry. But Elliott Vichinsky is concerned that the same problems that make current sickle-cell care ineffective will also plague this gene-therapy treatment. He estimates that at least 30% of his adult patients with sickle-cell disease die from preventable causes. As his patients attest, sickle-cell care is often inadequate for reasons that have little to do with scientific advancement and lots to do with economics and racism.