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.