One dose of gene therapy gives years of relief from blood disorder
Briefly

A recent study has highlighted the efficacy of gene therapy as a long-term treatment for haemophilia B, revealing a nearly tenfold decrease in bleeding episodes following a single dose. The positive outcomes of this treatment have been sustained for over 13 years, with no significant side effects reported, illustrating the potential for gene therapy to revolutionize care for men affected by this condition.
In a small study, gene therapy has demonstrated to be a safe long-term treatment for haemophilia B, showing a nearly tenfold reduction in average bleeding episodes after treatment.
The remarkable effects of a single dose of gene therapy persist for at least 13 years, indicating a significant advancement in the management of haemophilia B.
Read at Nature
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