公开(公告)号：US20240101972A1

公开(公告)日：2024-03-28

申请号：US18535857

申请日：2023-12-11

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Mustafa Yazicioglu ,  Ahmet Yunus Ozdemir

IPC: C12N7/00 ,  C07K14/005 ,  C12N9/06

CPC classification number: C12N7/00 ,  C07K14/005 ,  C12N9/003 ,  C12Y105/01003 ,  C07K2319/95 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14152

Abstract: The presently disclosed subject matter relates to compositions and methods for regulating recombinant adeno-associated virus (rAAV) production in cell culture. In particular, the presently disclosed subject matter relates to strategies to overcome AAV Rep protein-mediated cytotoxicity by reversible post-translational regulation of the expression of AAV Rep and helper proteins, resulting in regulated rAAV production.