公开(公告)号：US20220387560A1

公开(公告)日：2022-12-08

申请号：US17816367

申请日：2022-07-29

Applicant: SPARK THERAPEUTICS, INC.

Inventor： George Buchlis ,  Xavier Anguela ,  Katherine A. High

IPC: A61K38/46 ,  C12N9/22 ,  C12N15/10 ,  C12N15/864 ,  A61K31/7088

Abstract: Disclosed herein are compositions and methods of treating and/or correcting ocular disease in a subject, such as a mammal (e.g., human) eye using an Adeno-associated virus (AAV) system. The AAV system employs a nucleic acid encoding a CRISPR-Cas9 system for targeted gene disruption or correction.

公开(公告)号：US20190038724A1

公开(公告)日：2019-02-07

申请号：US16156649

申请日：2018-10-10

Applicant: SPARK THERAPEUTICS, INC.

Inventor： George Buchlis ,  Xavier Anguela ,  Katherine A. High

IPC: A61K38/46 ,  C12N15/10 ,  C12N9/22 ,  A61K31/7088

Abstract: Disclosed herein are compositions and methods of treating and/or correcting ocular disease in a subject, such as a mammal (e.g., human) eye using an Adeno-associated virus (AAV) system. The AAV system employs a nucleic acid encoding a CRISPR-Cas9 system for targeted gene disruption or correction.

公开(公告)号：US20160346359A1

公开(公告)日：2016-12-01

申请号：US15143272

申请日：2016-04-29

Applicant: Spark Therapeutics, Inc.

Inventor： George Buchlis ,  Xavier Anguela ,  Katherine A. High

IPC: A61K38/46 ,  A61K31/7088

CPC classification number: A61K38/465 ,  A61K31/7088 ,  A61K48/00 ,  C12N9/22 ,  C12N15/102 ,  C12N2750/14143

Abstract: Disclosed herein are compositions and methods of treating and/or correcting ocular disease in a subject, such as a mammal (e.g., human) eye using an Adeno-associated virus (AAV) system. The AAV system employs a nucleic acid encoding a CRISPR-Cas9 system for targeted gene disruption or correction.

Abstract translation: 本文公开了使用腺相关病毒（AAV）系统治疗和/或矫正受试者例如哺乳动物（例如人）眼睛的眼部疾病的组合物和方法。 AAV系统采用编码CRISPR-Cas9系统的核酸进行靶基因破坏或校正。