公开(公告)号：US20240102050A1

公开(公告)日：2024-03-28

申请号：US18355380

申请日：2023-07-19

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

Inventor： Rachel M. BAILEY ,  Steven J GRAY

IPC: C12N15/86 ,  A61K48/00 ,  A61P25/02 ,  C07K14/01 ,  C07K16/08

CPC classification number: C12N15/86 ,  A61K48/005 ,  A61P25/02 ,  C07K14/01 ,  C07K16/081

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and/or disorders in a subject, including, but not limited to neurological disorders such as giant axonal neuropathy. The methods described herein include direct administration of a gene therapy (e.g. an rAAV viral vector) to a subject via injection into a vagus nerve (e.g. the left vagus nerve) of the subject.

公开(公告)号：US20210139933A1

公开(公告)日：2021-05-13

申请号：US17092239

申请日：2020-11-07

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

Inventor： Qinglan LING ,  Steven J GRAY

IPC: C12N15/86 ,  A61P43/00

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SURF1 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 SURF1 polypeptide.