公开(公告)号：US12077772B2

公开(公告)日：2024-09-03

申请号：US17092240

申请日：2020-11-07

Applicant: THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM

Inventor： Steven J. Gray ,  Frances C. Shaffo

IPC: C12N15/86 ,  A61K9/00 ,  A61K38/17 ,  A61K48/00 ,  C07K14/705 ,  C12N7/00

CPC classification number: C12N15/86 ,  A61K9/0085 ,  A61K38/1787 ,  A61K48/005 ,  A61K48/0083 ,  C07K14/70571 ,  C12N7/00 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2830/50

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.