CCR5

On ERV, Abbie Smith provides an update on a pioneering treatment for hemophilia that uses viruses to insert missing genes in a patient's DNA. Hemophilia results from from the mutation or deletion of a gene that makes a blood clotting agent called Factor IX; without it, hemophiliacs are at risk for uncontrolled bleeding. While Factor IX can be delivered pharmaceutically, utilizing viruses to modify patients' DNA yields long-term improvements in natural Factor IX production. Abbie writes, "the amount of therapeutic Factor IX these patients needed (on average) dropped from 2613 IU/kg to 206. The…