Invention Grant
- Patent Title: Composition and methods for highly efficient gene transfer using AAV capsid variants
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Application No.: US14394454Application Date: 2013-04-18
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Publication No.: US09909142B2Publication Date: 2018-03-06
- Inventor: Mustafa N. Yazicioglu , Federico Mingozzi , Xavier Anguela , Katherine A. High
- Applicant: THE CHILDREN'S HOSPITAL OF PHILADELPHIA
- Applicant Address: US PA Philadelphia
- Assignee: The Children's Hospital of Philadelphia
- Current Assignee: The Children's Hospital of Philadelphia
- Current Assignee Address: US PA Philadelphia
- Agency: Pillsbury Winthrop Shaw Pittman LLP
- International Application: PCT/US2013/037170 WO 20130418
- International Announcement: WO2013/158879 WO 20131024
- Main IPC: C12N15/86
- IPC: C12N15/86 ; A61K48/00 ; C07K14/005

Abstract:
Compositions and methods for AAV mediated gene therapy are disclosed. AAV vectors comprise protein capsid variants comprising therapeutically beneficial transgenes. AAV variants are provided which exhibit increased transduction efficiency when compared to AAV serotypes (e.g., AAV1, AAV2, AAV8, AAV-rh74), which lack the modifications disclosed herein. Such improved vectors are useful for transduction of a variety of tissues.
Public/Granted literature
- US20150065562A1 COMPOSITION AND METHODS FOR HIGHLY EFFICIENT GENE TRANSFER USING AAV CAPSID VARIANTS Public/Granted day:2015-03-05
Information query
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