Therapy of genes prove effective for hemophilia

SAN DIEGO - Reuters

A single treatment with gene therapy, an experimental technique for fixing faulty genes, has been shown to boost output of a vital blood clotting factor, possibly offering a long-term solution for people with hemophilia B. Researchers said the same technology was also being studied as a treatment for hemophilia A, the far more common type of the inherited bleeding disorder. “It is a technique for potentially permanently curing patients,” said Dr. Charles Abrams, American Society of Hematology secretary and associate chief of hematology/oncology at the University of Pennsylvania in Philadelphia. Both safety and efficacy have held back the field of gene therapy. One experiment cured two French boys with a rare immune disorder but gave them leukemia in 2002, and an Arizona teenager died in a 1999 gene therapy experiment. The approach used by researchers at the University College London and St. Jude Children’s Research Hospital in Memphis, involved the use of a novel viral “vector.”