COWAN, CHAD ALBERT,KABADI, AMI MEDA,LUNDBERG, ANTE SVEN
申请号：
CA3009308
公开号：
CA3009308A1
申请日:
2016.12.22
申请国别(地区):
CA
年份:
2017
代理人:
摘要：
The present application provides materials and methods for treating a patient with Amyotrophic Lateral Sclerosis (ALS) and/or Frontaltemporal Lobular Degeneration (FTLD), both ex vivo and in vivo. In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the C9ORF72 gene in a cell by genome editing.
