THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY; INC.;BOCO SILICON VALLEY;BOCO SILICON VALLEY, INC.
发明人:
PORTEUS, MATTHEW H.,KILDEBECK, ERIC J.,DEVER, DANIEL P.,CLARK, JOSEPH T.,TSUKAMOTO, ANN,UCHIDA, NOBUKO
申请号：
CA3020181
公开号：
CA3020181A1
申请日:
2017.04.13
申请国别(地区):
CA
年份:
2017
代理人:
摘要：
The invention provides methods for generating a genetically modified human neural stem cell, genetically modified human neural stem cells, and pharmaceutical compositions comprising the genetically modified human neural stem cells. In particular said methods involve the use of a DNA nuclease, such as TALEN, ZFN or CRISPR/Cas mediated genome editing. Also provided are associated kits. The invention also provides methods for preventing or treating a neurodegenerative disease or a neurological injury in a human subject using genetically modified human neural stem cells.
