Researchers have reported a major breakthrough in gene therapy that could transform the treatment of a rare blood disorder affecting thousands of patients worldwide.
The therapy, tested on 20 patients, showed significant improvement in 17 patients after just six months. Scientists say the approach corrects the underlying genetic defect rather than simply managing symptoms.
"This is the most encouraging result we have seen in two decades of work on this condition," said the study's lead investigator. Researchers cautioned that longer follow-up is needed before the therapy can be approved for wider use.
The trial was conducted across four centres and followed participants for a median of 11 months. Adverse events were described as mild and transient.
