China’s thalassaemia jab raises hope for common inherited blood disorder
Chinese researchers have developed a new gene therapy for thalassaemia, a common inherited blood disorder. The treatment involves modifying a patient's own stem cells to produce functional haemoglobin. Early clinical trials show promising results, offering hope for a potential cure.
- ▪Thalassaemia is a genetic blood disorder that impairs haemoglobin production.
- ▪The new therapy uses CRISPR gene-editing technology to correct mutations in blood stem cells.
- ▪Patients in the trial were able to produce normal haemoglobin and reduce or eliminate the need for blood transfusions.
- ▪The treatment represents a significant advancement in gene therapy for inherited diseases.
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Excerpt limited to ~120 words for fair-use compliance. The full article is at South China Morning Post.