Sickle cell mice cured by stem cells reprogrammed from their own tails

Stem cells have long been hyped as the shiny future of medicine. Their ability to produce to every type of cell in the body provided hope for treating diseases from Alzheimer’s, to Parkinson’s to stroke, by providing a ready supply of replacement cells. Despite years of slow progress, we are now tantalisingly close to turning this hype into reality and a new study suggests that the dawn of promised stem cell treatments is getting closer.

For the first time, scientists have cured mice of a genetic disorder called sickle cell anaemia using personalised stem cells reprogrammed from cells in their tails. The study is a powerful ‘proof-of-principle’ that reprogrammed stem cells could one day fulfil their potential in fighting human disease.

The personal touch is of the utmost importance. It’s no good just giving someone any old stem cells. Genetic differences between the donor and recipient could cause problems in the long-term and trigger attack and rejection from the hosts’ immune system in the short-term. The trick is to convert a patient’s own cells into personalised stem cells for their own private use.

Last year, a group of Japanese scientists found a way to do this in mice and produced “induced pluripotent stem cells” (iPSCs) that were very similar to embryonic stem cells. And just last month, I blogged about two breakthrough papers which showed that human cells could also be reprogrammed into iPSCs.

Now, Jacob Hanna and colleagues from the Whitehead Institute for Biomedical Research, the University of Alabama and MIT, have used these reprogrammed cells to cure a genetic disease – sickle cell anaemia.