Research Institute at Nationwide Children's Hospital;Research Institute at Nationwide Childrens Hospital
发明人:
Rodino-Kalapac, Louise,Potter, Rachael
申请号：
AU2016358172
公开号：
AU2016358172A1
申请日:
2016.11.15
申请国别(地区):
AU
年份:
2018
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.
