Patents Assigned to MURDOCH CHILDENS RESEARCH INSTITUTE
  • METHOD OF TREATMENT
    Publication number: 20140031412
    Abstract: The present disclosure teaches the treatment of a blood pathology, such as a blood pathology associated with impaired hemoglobin synthesis including the treatment of ?-thalassemia or a related hemoglobinopathy. An RNA molecule such as a short interfering RNA or a hairpin RNA which targets an mRNA species encoding ?-globin is administered to a subject to reduce the amount of ?-globin produced to non-zero levels and ameliorate the effects of an ?- and ?-globin chain imbalance.
    Type: Application
    Filed: December 20, 2011
    Publication date: January 30, 2014
    Applicant: MURDOCH CHILDENS RESEARCH INSTITUTE
    Inventor: Jim Vadolas