公开(公告)号：US20250011791A1

公开(公告)日：2025-01-09

申请号：US18709305

申请日：2022-11-17

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

Inventor： Berge A. MINASSIAN ,  Emrah GUMUSGOZ ,  Mayank VERMA

IPC: C12N15/113 ,  A61P3/00 ,  A61P25/28 ,  C12N15/86

Abstract: The present disclosure provides methods and compositions for the treatment of glycogen storage disorders, such as, for example, Lafora Disease and adult polyglucosan body disease. The methods and compositions of the present disclosure comprise isolated nucleic acid molecules, rAAV vectors and rAAV viral vectors comprising polynucleotide sequences encoding for artificial micro RNAs (amiRNAs) directed against GYS1.