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.


How cerebral organoids are guiding brain-cancer research and therapies – Nature

Image credit: M. Lancaster/MRC-LMB

People with glioblastoma multiforme, one of the most common forms of brain cancer, have a median survival of less than 15 months after diagnosis. If researchers could grow numerous small brain-like structures that contained a replica of the person’s tumour and then bathe them in various treatments, in the space of a few weeks, they might learn exactly which ones would have the best chance of fighting brain cancer in that individual. Howard Fine, a neuro-oncologist at Weill Cornell Medicine in New York City, is developing such models, known as cerebral organoids. Organoids are particularly valuable for studying brain cancer because neither human brain tumours transplanted into mice nor human tumour stem cells grown in a culture dish behave in the same way as their counterparts in the body.