公开(公告)号：US20210139934A1

公开(公告)日：2021-05-13

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 ,  C12N7/00 ,  C07K14/705 ,  A61K38/17 ,  A61K48/00 ,  A61K9/00

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.

公开(公告)号：US20240376498A1

公开(公告)日：2024-11-14

申请号：US18785836

申请日：2024-07-26

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

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.