Original therapy integrating stem cell tech and gene editing could treat a blood disorder with 350 million carriers worldwide
Thalassaemia is the most common genetic blood disorder in the world. China currently has 30 million thalassaemia gene carriers, while the global total reaches 350 million.
Severe thalassaemia patients could confront a stark reality: regular blood transfusions, potential organ transplants, even the risk of premature death.
If two carriers of the gene marry, their children have a 25 per cent chance of developing the disease.
The therapy used CS-101, a base-editing drug developed for thalassaemia patients by scientists at ShanghaiTech University using their transformer Base Editor (tBE) technology.
Five Chinese patients with beta-thalassaemia who received CS-101 treatment rapidly restored haematopoietic function – the ability to produce healthy new blood cells. Total haemoglobin and fetal haemoglobin (HbF) levels rose quickly and stayed high, freeing them from blood transfusions.
Source: News - South China Morning Post
