MATERIALS AND METHODS FOR TREATMENT OF FRIEDREICH ATAXIA AND OTHER RELATED DISORDERS

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专利权人:

发明人:

申请号：

EP17742544.4

公开号：

EP3478828A1

申请日:

2017.22.06

申请国别(地区):

EP

年份:

2019

代理人:

摘要：

The present application provides materials and methods for treating a patient with one or more condition associated with FXN whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of FXN gene in a cell by genome editing.

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