DUAL AAV VECTOR SYSTEM FOR CRISPR/CAS9 MEDIATED CORRECTION OF HUMAN DISEASE

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专利权人:

THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

发明人:

WILSON, James, M.,WANG, Lili,YANG, Yang

申请号：

WO2016US29330

公开号：

WO2016176191(A1)

申请日:

2016.04.26

申请国别(地区):

世界知识产权组织国际局

年份:

2016

代理人:

摘要：

A dual vector expression system is provided for delivery of CRISPR/Cas9, sgRNA and donor templates for supplying corrected genes or gene sequences to neonatal subjects. An AAV8 system for delivery to proliferating cells such as hepatocytes is described, as are other dual AAV vector systems for targeting to other cells.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/