公开(公告)号：US20240350667A1

公开(公告)日：2024-10-24

申请号：US18685681

申请日：2022-07-22

Applicant: Spark Therapeutics, Inc.

Inventor： Xavier Anguel ,  Pedro Cejas ,  Ali Nahvi ,  Mustafa Yazicioglu, III ,  Rui Zhang

IPC: A61K48/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/404 ,  A61K31/437 ,  A61K45/06 ,  C12N15/88

CPC classification number: A61K48/005 ,  A61K9/127 ,  A61K9/5123 ,  A61K31/404 ,  A61K31/437 ,  A61K45/06 ,  A61K48/0083 ,  C12N15/88

Abstract: The present invention features method and composition that can be used to facilitate intracellular delivery of DNA to a subject. The provided methods and compositions employ a nanoparticle for intracellular DNA delivery and a cytosolic DNA-sensing inhibitor. The cytosolic DNA-sensing inhibitor is provided to decrease the subject's immune response that can be stimulated by the DNA.