Thomas James CRADICK,Chad Albert COWAN,Ante Sven LUNDBERG,Gregory Joseph COST
申请号：
US16074743
公开号：
US20190038771A1
申请日:
2017.02.02
申请国别(地区):
US
年份:
2019
代理人:
摘要：
The present application provides materials and methods for treating a patient with severe combined immunodeficiency (SCID) or Omenn Syndrome, both ex vive and in vive. In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the Recombination Activating Gene 1 (RAG1) gene in a cell by genome editing.
