Invention Grant
- Patent Title: rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
-
Application No.: US15550864Application Date: 2016-02-16
-
Publication No.: US10648000B2Publication Date: 2020-05-12
- Inventor: William W. Hauswirth , Sanford L. Boye , Daniel M. Lipinski , Michael E. Boulton , Shannon E. Boye
- Applicant: University of Florida Research Foundation, Incorporated
- Applicant Address: US FL Gainesville
- Assignee: University of Florida Research Foundation, Incorporated
- Current Assignee: University of Florida Research Foundation, Incorporated
- Current Assignee Address: US FL Gainesville
- Agency: Wolf, Greenfield & Sacks, P.C.
- International Application: PCT/US2016/018098 WO 20160216
- International Announcement: WO2016/133917 WO 20160825
- Main IPC: C12N15/86
- IPC: C12N15/86 ; C12N9/78 ; A61K38/50 ; A61K48/00 ; C07K14/005 ; C07K14/705 ; C12N7/00 ; A61K38/00

Abstract:
Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian vascular system, and complications from Type I diabetes. Also disclosed are methods for systemic and tissue-localized delivery of therapeutic rAAV-based gene expression cassettes to vascular endothelial cells, tissues, and organs, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of vasculitis, and complications arising from Type I diabetes, such as macular edema, nephropathy, diabetic retinopathy, and the like.
Public/Granted literature
Information query
IPC分类: