Tag Archives: Gene therapy

“Bubble Boys” Lose Bubbles Thanks to Gene Therapy


“Bubble Boys” Lose Bubbles Thanks to Gene Therapy

Historically, patients with severe combined immunodeficiency (SCID), an often fatal congenital disorder in which the cells involved in immune responses fail to work properly, have been kept in isolation in a sterile environment to protect them from infection. Currently, the only long-term treatment for so-called bubble boy disease is a bone marrow transplant, but an experimental gene therapy is showing promise for boys with X-linked SCID. Of the nine babies given the treatment, six developed immune systems capable of fighting off infection. More… Discuss

Scientists Create Biological Pacemaker


Scientists Create Biological Pacemaker

Scientists have successfully tested in pigs a form of gene therapy that turns ordinary cardiac muscle cells into specialized ones that signal to the heart when it should beat, effectively creating a biological pacemaker that restored normal heart rhythm to pigs with heart block. Pig and human hearts are quite similar, so the results are encouraging, and researchers hope to begin human trials within a few years. Eventually, this procedure could offer an alternative to artificial pacemakers, which require surgical implantation and regular replacement. More…