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takyon [soylentnews.org] writes:

A gene therapy trial has shown success in reducing bleeding episodes [sciencemag.org] caused by the inherited blood disorder hemophilia B [wikipedia.org]. Ten participants received a virus that stimulated the production of the blood-clotting protein factor IX [wikipedia.org]. Nine have had no bleeding episodes, while eight no longer need the gene therapy injections every few days:

Previous gene therapy trials for hemophilia B didn't go well, either because patients' immune systems destroyed the modified cells or the cells didn't make enough factor IX. In the new trial, sponsored by Spark Therapeutics and Pfizer, researchers gave the patients' liver cells the gene for an unusually potent version of the factor IX protein. That allowed the team to lower the vector dose, minimizing immune responses. Two patients had elevated liver enzymes in reaction to the vector, but those levels came down after they received steroids. Only 20% of hemophilia patients have the B form, but efforts are also underway to use gene therapy to treat the most common type, hemophilia A.

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant [nejm.org] (DOI: 10.1056/NEJMoa1708538) (DX [doi.org])

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