IP Library Granted Patent US 10,172,880
Granted Patent B2
US 10,172,880 · App. 15/182,773 · Granted Jan 8, 2019

Talen-based gene correction

Inventors: Mark Osborn (Minneapolis, MN); Jakub Tolar (Minneapolis, MN); Bruce Robert Blazar (Golden Valley, MN); Daniel Voytas (Falcon Heights, MN)
Assignee: Regents of the University of Minnesota
A61K31/713A61K31/7088C12N9/16
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Quick Facts
Patent No.
US 10,172,880
App. No.
15/182,773
Granted
Jan 8, 2019
Kind
B2
Abstract

The invention is directed to transcription activator-like effector nuclease (TALEN)-mediated DNA editing of disease-causing mutations in the context of the human genome and human cells to treat patients with compromised genetic disorders.

Claims (10)

1. A method to treat a genetic disease or disorder caused by a genetic mutation in a target gene comprising: contacting a cell with

a composition comprising a nucleic acid encoding at least one TALEN protein, wherein the at least one TALEN protein is capable of inducing a site-specific double stranded DNA break in a target gene in a cell, wherein the target gene is a COL7A1 gene; and

a nucleic acid donor sequence,

wherein the donor sequence is a template for correction of a genetic mutation in the Col7A1 target gene, and further wherein the genetic mutation is capable of causing epidermolysis bullosa,

thereby treating the genetic disease or disorder caused by the genetic mutation in the target gene.

2. The method of claim 1 , wherein the cell is selected from the group consisting of a fibroblast, keratinocyte, inducible pluripotent stem cell, hematopoietic stem cell, mesenchymal stem cell, embryonic stem cell, hematopoietic progeny cell, T-cell, B-cell, glial cell, neural cell, neuroglial progenitor cell, neuroglial stem cell, muscle cell, lung cell, pancreatic cell, liver cell and a cell of the reticular endothelial system.

3. The method of claim 1 , wherein the composition comprises a nucleic acid which encodes a first TALEN protein which is a left TALEN and the composition compromises a nucleic acid which encodes a second TALEN which is a right TALEN that cooperates with the left TALEN to make a site-specific double stranded DNA break in the target gene.

4. The method of claim 1 , wherein the nucleic acid encoding the TALEN or the nucleic acid donor sequence is part of a vector or plasmid.

5. The method of claim 3 , wherein the first TALEN and/or the second TALEN comprise a plurality of TAL effector repeat sequences and the endonuclease domain and a spacer between the plurality of TAL effector repeat sequences and the endonuclease domain includes a spacer.

6. The method of claim 5 , wherein the spacer is 12 to 30 nucleotides in length.

Assignments (2)
CONFIRMATORY LICENSE Recorded May 11, 2017
From: UNIVERSITY OF MINNESOTA
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 042444/0351 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 18, 2016
From: OSBORN, MARK J.; TOLAR, JAKUB; VOYTAS, DANIEL F.; BLAZAR, BRUCE R.
To: REGENTS OF THE UNIVERSITY OF MINNESOTA
Reel/Frame 039176/0491 →
Continuity (3)
Division 14193037 · Feb 28, 2014
Provisional Application 61771735 · Mar 1, 2013
Related Publication 20160367588A1 · Dec 22, 2016
Cited By (1)
US 12,319,925