Invention Application
- Patent Title: METHODS OF TREATING MITOCHONDRIAL DISORDERS
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Application No.: US17484324Application Date: 2021-09-24
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Publication No.: US20220009977A1Publication Date: 2022-01-13
- Inventor: Stephanie Cherqui
- Applicant: The Regents of the University of California
- Applicant Address: US CA Oakland
- Assignee: The Regents of the University of California
- Current Assignee: The Regents of the University of California
- Current Assignee Address: US CA Oakland
- Main IPC: C07K14/47
- IPC: C07K14/47 ; C12N15/86 ; A61K35/28 ; A01K67/027 ; A61P25/28 ; C12N9/22 ; C12N15/11

Abstract:
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.
Public/Granted literature
- US12012437B2 Methods of treating mitochondrial disorders Public/Granted day:2024-06-18
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