Invention Grant
- Patent Title: Transgene cassettes, AAV vectors and AAV viral vectors for the expression of human codon-optimized SLC6A1
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Application No.: US17092240Application Date: 2020-11-07
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Publication No.: US12077772B2Publication Date: 2024-09-03
- Inventor: Steven J. Gray , Frances C. Shaffo
- Applicant: THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
- Applicant Address: US TX Austin
- Assignee: THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
- Current Assignee: THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
- Current Assignee Address: US TX Austin
- Agency: DLA Piper LLP US
- Main IPC: C12N15/86
- IPC: C12N15/86 ; A61K9/00 ; A61K38/17 ; A61K48/00 ; C07K14/705 ; C12N7/00
Abstract:
The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SLC6A1 loss and/or misfunction. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a GAT1 polypeptide.
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