The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Oct. 24, 2023
Filed:
May. 31, 2018
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
University of Southern California, Los Angles, CA (US);
The Research Institute of the Mcgill University Health Centre, Montreal, CA;
Nancy Braverman, Montreal, CA;
Catherine Argyriou, Montreal, CA;
Joseph Hacia, Pasadena, CA (US);
Jean Bennett, Philadelphia, PA (US);
Junwei Sun, Philadelphia, PA (US);
Ji Yun Song, Lower Gwynedd, PA (US);
Devin McDougald, Philadelphia, PA (US);
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
University of Southern California, Los Angeles, CA (US);
The Research Institute of McGill University Health Centre, Montreal, CA;
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.