Patients living with sickle cell disease may one day find respite in gene editing, a new study from the US reveals. Sickle cell disease (SCD) is a genetic disorder that affects the haemoglobin - oxygen-carrying molecules in our blood - of hundreds of thousands of people worldwide. The disease can lead to anaemia, pain, organ failure and stroke, and SCD patients have a lifespan of just 40 to 60 years. Now, a potential new treatment could be in the works, thanks to a development in genetic editing. Mark DeWitt from the University of California, Berkeley, and colleagues used a precise technique in genetic modification called CRISPR-Cas9 to alter the problem cells that cause SCD. CRISPR is a process
A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients. For the first time, they have corrected the mutation in a proportion of stem cells that is high enough to produce a substantial benefit in sickle cell patients. The researchers from the University of California, Berkeley, UCSF Benioff Children's Hospital Oakland Research Institute (CHORI) and the University of Utah School of Medicine hope to re-infuse patients with the edited stem cells and alleviate symptoms of the disease, which primarily afflicts those of African descent and leads to anemia, painful blood blockages and early death.
The promise of a revolutionary gene-editing technology is beginning to be realized in experiments aimed at curing sickle cell disease. Scientists reported Wednesday that they used the CRISPR-Cas9 system to correct a tiny genetic mutation that causes the blood disease, which affects millions of people around the world. So far, the feat has been demonstrated only in human cells that were confined to laboratory dishes. But in a promising step, the researchers used the same DNA-editing technique to alter human cells that were transfused into mice. After 16 weeks, the mice still had cells that contained the edited gene, according to a study in the journal Science Translational Medicine. “What we