The Research Institute at Nationwide Children's Hospital
发明人:
RODINO-KALAPAC, Louise,POTTER, Rachael
申请号：
HK19100317
公开号：
HK1257955A1
申请日:
2019.01.09
申请国别(地区):
HK
年份:
2019
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.
