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Gene therapy study offers proof of potential breakthrough on hemophilia

from FierceBiotech

A transatlantic team of investigators has presented some promising — though very early stage — breakthrough data on a new gene therapy for hemophilia B. The investigators say that their gene therapy program came close to curing four of six patients, spurring the body to produce enough clotting factor to eliminate the need for regular protein injections. And the other two were helped by the treatment, with a reduced need for injections. more


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