RESEARCH INSTITUTE OF NATIONWIDE CHILDRENS HOSPITAL;RESEARCH INSTITUTE OF NATIONWIDE CHILDREN'S HOSPITAL
发明人:
RODINO-KALPAC, LOUISE,POTTER, RACHAEL
申请号：
CA3005633
公开号：
CA3005633A1
申请日:
2016.11.15
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 a titin-based myopathy, particularly a titin-based cardiomyopathy, and/or other titinopathy. In addition, the present application provides materials and methods for editing the titin gene in a cell by genome editing.
