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METHODS OF TREATING MITOCHONDRIAL DISORDERS
专利权人:
The Regents of the University of California
发明人:
Stephanie Cherqui
申请号:
US16820368
公开号:
US20200206361A1
申请日:
2020.03.16
申请国别(地区):
US
年份:
2020
代理人:
摘要:
Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.
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