The children of sickle cell disease are growing up – Nature

Taj Francis

Sickle-cell disease was once a childhood ailment, simply because many children with the condition died before reaching adulthood. Since 1972, the United States has managed to drastically reduce childhood deaths from the disease, but the outlook has worsened for adults. 

“Adult sickle-cell programmes should be funded like the paediatric ones are,” says Sophie Lanzkron, a haematologist specializing in adults at the Johns Hopkins University School of Medicine in Baltimore, Maryland. It’s not about education: specialists generally know what to do, the health-care system just isn’t doing it, she says.

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.

 

Gene-Editing Startup Raises $120 Million to Apply CRISPR to Medicine

Editas Medicine, a company at the forefront of developing the gene-editing technology known as CRISPR, has raised $120 million to create new treatments for conditions including cancer, retinal disease, and sickle-cell anemia.

Monday’s announcement reflects a surge of interest in CRISPR, a technology that is only a few years old. It also serves to clarify the goals and strategy of Editas, which was founded by some of the most prominent inventors of the gene-editing system, including Feng Zhang, a researcher at the Broad Institute of Harvard and MIT.

Read the full article in MIT Technology Review.