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.

Date of Patent:
Jul. 18, 2023

Filed:

May. 26, 2021
Applicant:

Institut National DE LA Sante ET DE LA Recherche Medicale (Inserm), Paris, FR;

Inventors:

Marisol Corral-Debrinski, Montreuil, FR;

Jose-Alain Sahel, Paris, FR;

Valerie Kaltimbacher, Fontenay Sous Bois, FR;

Crystel Bonnet, Montrouge, FR;

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/85 (2006.01); C12N 15/00 (2006.01); A61K 48/00 (2006.01); C12N 9/02 (2006.01); C07K 14/47 (2006.01); C12N 9/10 (2006.01);
U.S. Cl.
CPC ...
C12N 15/85 (2013.01); A61K 48/005 (2013.01); A61K 48/0058 (2013.01); A61K 48/0066 (2013.01); C07K 14/47 (2013.01); C12N 9/0053 (2013.01); C12N 9/0089 (2013.01); C12N 9/1085 (2013.01); C12N 15/8509 (2013.01); C12Y 109/03001 (2013.01); C07K 2319/07 (2013.01); C12N 2800/22 (2013.01); C12N 2810/80 (2013.01);
Abstract

An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3'UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3′UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.


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