Patents Assigned to The Research Institute of McGill University Health Centre
  • Gene therapy for treating peroxisomal disorders
    Patent number: 11793887
    Abstract: Methods and compositions are provided for treatment of peroxisomal biogenesis disorders (PBDs). More particularly, recombinant adeno-associated viruses (rAAV) provided in the form of compositions are used to deliver a nucleic acid encoding human PEX1 to host cells. The rAAVs comprise a AAV capsid, and packaged therein a vector genome comprising an AAV 5? inverted terminal repeat (ITR) sequence; a promoter; a coding sequence encoding a human PEX1; and an AAV 3? ITR.
    Type: Grant
    Filed: May 31, 2018
    Date of Patent: October 24, 2023
    Assignees: The Trustees of the University of Pennsylvania, University of Southern California, The Research Institute of McGill University Health Centre
    Inventors: Nancy Braverman, Catherine Argyriou, Joseph Hacia, Jean Bennett, Junwei Sun, Ji Yun Song, Devin McDougald