COWAN CHAD ALBERT,KABADI AMI MEDA,LUNDBERG ANTE SVEN
申请号：
IN201847026630
公开号：
IN201847026630A
申请日:
2018.07.17
申请国别(地区):
IN
年份:
2018
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.
