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.

公开(公告)号：US20240344084A1

公开(公告)日：2024-10-17

申请号：US18603950

申请日：2024-03-13

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

Inventor： Berge A. MINASSIAN ,  Emrah GUMUSGOZ

IPC: C12N15/86 ,  C12N7/00

CPC classification number: C12N15/86 ,  C12N7/00 ,  C12N2750/14143 ,  C12N2750/14151

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to CSTB loss and/or misfunction. The methods and compositions of the present disclosure include rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules encoding CSTB polypeptides.

公开(公告)号：US20240318198A1

公开(公告)日：2024-09-26

申请号：US18274485

申请日：2022-02-02

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

Inventor： Ryan BUTLER ,  Steven J. GRAY ,  Hye Ri KANG ,  Berge A. MINASSIAN ,  Emrah GUMUSGOZ

IPC: C12N15/86 ,  C12N15/113

CPC classification number: C12N15/86 ,  C12N15/113 ,  C12N2310/531 ,  C12N2750/14143

Abstract: The present disclosure provides methods and compositions for the treatment of Angelman Syndrome The methods and compositions of the present disclosure comprise isolated nucleic acid molecules, rAAV vectors and rAAV viral vectors comprising polynucleotide sequences encoding for short hairpin RNA (shRNA) molecules directed against UBE3A-ATS.