公开(公告)号：US20190022192A1

公开(公告)日：2019-01-24

申请号：US15574420

申请日：2016-04-15

Applicant: Genzyme Corporation

Inventor： Guoxiang RUAN ,  Abraham SCARIA

IPC: A61K38/46 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90 ,  A61K31/7105 ,  A61K35/761

Abstract: Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non-naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self-limiting CRISPER-Cas system.

公开(公告)号：US20160068844A1

公开(公告)日：2016-03-10

申请号：US14785159

申请日：2014-04-17

Applicant: GENZYME CORPORATION ,  THE UNITED STATES OF AMERICA,AS REPRESENTED BY THE SECRETARY,DEPARTMENT OF HEALTH AND HUMAN SERVICE

Inventor： Samuel WADSWORTH ,  Abraham SCARIA ,  Chi-Chao CHAN

IPC: C12N15/113

CPC classification number: C12N15/1136 ,  A61K38/1793 ,  C07K14/7155 ,  C07K2319/30 ,  C12N2750/14143

Abstract: Compositions and methods for treating macular degeneration are disclosed. The methods utilize IL17 inhibitors, such as IL17 receptors, as well as fusion proteins including an IL17 receptor fused with a multimerization domain, and recombinant viral vectors encoding such fusions.

Abstract translation: 公开了治疗黄斑变性的组合物和方法。 所述方法利用IL17抑制剂，例如IL17受体，以及融合蛋白，包括与多聚化结构域融合的IL17受体，以及编码这种融合物的重组病毒载体。