SCMP Economy28.04.2026Ciencia1 dk okumaChina

Chinese Scientists Develop Gene Therapy CS-101 Curing Five Beta-Thalassaemia Patients

Base-editing drug using transformer Base Editor technology frees patients from blood transfusions

En resumen

Scientists at ShanghaiTech University have developed CS-101, a base-editing gene therapy that successfully cured five Chinese patients with beta-thalassaemia.
The treatment restored haematopoietic function, raising total haemoglobin and fetal haemoglobin levels, freeing patients from lifelong blood transfusions.
China has 30 million thalassaemia gene carriers while the global total reaches 350 million.

Resumen generado por IA

Por qué importa

Thalassaemia is the world's most common genetic blood disorder. Patients with severe forms require regular blood transfusions and face potential organ transplants and premature death. When two carriers of the gene marry, their children have a 25 per cent chance of developing the disease.

Tamaño de fuente

Thalassaemia is the world's most common genetic blood disorder. China currently has 30 million thalassaemia gene carriers, while the global total reaches 350 million. Patient with severe thalassaemia could confront a stark reality: regular blood transfusions, potential organ transplants, and 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 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.