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US 9,850,497 B2Granted
Gene targeting methods and tools
AssigneeRegents of the University of Minnesota
InventorsRita Perlingeiro, Michael Kyba, Fabrizio Rinaldi, Daniel F. Voytas
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Abstract
Provided herein is a general method of correcting a gene, for example a gene with many known mutations, such as a gene with mutations in many different exons which could vary from subject to subject (e.g., patient), as well as a set of tools (TALENs and gene targeting vectors) to accomplish such method.
Claims6
An in vitro method to correct a dysfunctional gene, wherein the dysfunction is caused by a genetic mutation, the method comprising:
- introducing one or more nucleic acids encoding a TALEN and a nucleic acid donor sequence into a cell comprising said dysfunctional gene, wherein the TALEN comprises a plurality of TAL effector sequences that can bind to a target site of insertion in the dysfunctional gene,
- wherein said nucleic acid donor sequence comprises a stop codon and a nucleic acid encoding a wild-type, functional coding region of said dysfunctional gene flanked by sequences homologous to the target site of insertion, wherein the cell expresses said TALEN and said TALEN introduces a site-specific double stranded break at the target site of insertion in said dysfunctional gene, thereby introducing a stop codon into the dysfunctional gene in the cell and introducing a wild-type, functional coding region of said dysfunctional gene into said cell.
Dependencies are inferred from the claim text.