公开(公告)号：US20210363192A1

公开(公告)日：2021-11-25

申请号：US17050362

申请日：2019-04-26

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Xavier ANGUELA ,  Sean ARMOUR ,  Nicholas KEISER ,  Suryanarayan SOMANATHAN ,  Mustafa N. YAZICIOGLU

IPC: C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N15/113

Abstract: The invention provides modified adeno-associated virus (AAV) capsid proteins. Modified AAV capsid proteins include, for example, capsid proteins modified to have a peptide insertion comprising a nuclear localization signal (NLS) sequence, capsid proteins modified to have an amino acid substitution at an RXXL site or a (L/P)PXY site, where X can be any amino acid, and capsid proteins modified to have one or more particular amino acid positions substituted with a different amino acid.