The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Jun. 18, 2024
Filed:
Mar. 16, 2020
Applicant:
The Regents of the University of California, Oakland, CA (US);
Inventor:
Stephanie Cherqui, La Jolla, CA (US);
Assignee:
The Regents of the University of California, Oakland, CA (US);
Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); A61K 35/14 (2015.01); A61K 35/28 (2015.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/85 (2006.01);
U.S. Cl.
CPC ...
A61K 48/005 (2013.01); A61K 35/14 (2013.01); A61K 35/28 (2013.01); C12N 9/22 (2013.01); C12N 15/111 (2013.01); C12N 15/85 (2013.01); C12N 2310/20 (2017.05); C12N 2800/80 (2013.01);
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.