The Board of Trustees of the Leland Stanford Junior University; Inc.;BOCO Silicon Valley;BOCO Silicon Valley, Inc.
发明人:
Clark, Joseph T.,Porteus, Matthew H.,Kildebeck, Eric J.,Dever, Daniel P.,Tsukamoto, Ann,Uchida, Nobuko
申请号：
AU2017250683
公开号：
AU2017250683A1
申请日:
2017.04.13
申请国别(地区):
AU
年份:
2018
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.
