公开(公告)号：US20210180085A1

公开(公告)日：2021-06-17

申请号：US17112299

申请日：2020-12-04

Applicant: The Board of Regents of the University of Texas System

Inventor： Steven J. GRAY ,  Sarah Sinnett

IPC: C12N15/86 ,  C12N15/113

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to MeCP2 loss and/or misfunction, including RETT syndrome. 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 MeCP2 polypeptide.

公开(公告)号：US11891616B2

公开(公告)日：2024-02-06

申请号：US17112299

申请日：2020-12-04

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

Inventor： Steven J. Gray ,  Sarah Sinnett

IPC: C12N15/86 ,  C12N15/113

CPC classification number: C12N15/86 ,  C12N15/113 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/321

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to MeCP2 loss and/or misfunction, including RETT syndrome. 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 MeCP2 polypeptide.