DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE

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专利权人:

发明人:

James M. Wilson,Lili Wang,Yang Yang

申请号：

US15566150

公开号：

US20180110877A1

申请日:

2016.04.26

申请国别(地区):

US

年份:

2018

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.

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