Muscular dystrophy ‘death sentence’ targeted by gene therapy trials – Genetic Literacy Project
Genetic Literacy Project |
Muscular dystrophy ‘death sentence’ targeted by gene therapy trials
Genetic Literacy Project [T]hree U.S. teams say they are ready to try to treat Duchenne [Muscular Dystrophy] with gene therapy. The first study could begin as soon as next month at Nationwide Children’s Hospital in Columbus, Ohio… Two other tests on children…are slated to … |
Kaiser Health News |
‘Breakthrough’ Leukemia Drug Also Portends ‘Quantum Leap’ In Cost
Kaiser Health News A nurse reaches for blood samples taken from a patient receiving a kind of immunotherapy known as CAR T-cell therapy at the Fred Hutchinson Cancer Research Center in Seattle. Immune therapy is the hottest trend in cancer care. Its next frontier is … |
pharmaphorum |
Orchard’s gene therapy gains UK ‘promising innovative medicine’ status
pharmaphorum A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy. Orchard Therapeutics was only set up two years ago, but has now gained the PIM status from the UK’s medicines … |
MIT Technology Review |
Sickle-Cell Patients See Hope in CRISPR
MIT Technology Review The idea is that CRISPR could correct the genetic mutation responsible for sickle-cell so that patients’ bodies could make normal red blood cells, alleviating the pain and other severe symptoms associated with the disease. Researchers have already … |