# US 11,898,176 B2 · Genetic correction of mutated genes

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- Number: US 11,898,176 B2
- Document ID: US11898176B2
- Status: Granted
- Kind: B2
- Application number: 16/890,232
- Filed: 2020-06-02
- Published: 2021-02-11
- Granted: 2024-02-13
- Earliest claimed: 2012-04-27
- Assignees: Duke University
- Applicants: Duke University
- Inventors: Charles Gersbach; David Ousterout
- Web page: https://patentgrep.com/patent/US11898176B2

## CPC

- C12N 9/22: Ribonucleases {[RNase]; Deoxyribonucleases [DNase]}
- A61K 48/005: {characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered}
- C07K 14/4708: {Duchenne dystrophy}
- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- C12N 15/907: {in mammalian cells}
- C12N 2310/20: involving clustered regularly interspaced short palindromic repeats [CRISPR]
- C12N 2320/30: Special therapeutic applications
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)

## Abstract

Disclosed herein are transcription activator-like effector nuclease (TALEN)-related compositions and methods of using said TALENs for correcting mutant genes.

## Claims

### Claim 1

An isolated polynucleotide comprising a nucleotide sequence encoding an engineered transcription activator-like effector nuclease (TALEN) protein that binds to a dystrophin gene, wherein the TALEN protein binds upstream from a premature stop codon on the dystrophin gene, downstream from a premature stop codon on the dystrophin gene, a region in exon 51 of the dystrophin gene, or a region in the 5′ UTR of the dystrophin gene, and wherein the TALEN protein comprises an amino acid sequence selected from the group consisting of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO: 5, SEQ ID NO: 6, SEQ ID NO: 7, SEQ ID NO: 8, SEQ ID NO: 9, SEQ ID NO: 10, SEQ ID NO: 11, SEQ ID NO: 12, SEQ ID NO: 13, SEQ ID NO: 14, and SEQ ID NO: 15.

### Claim 2 (depends on 1)

The isolated polynucleotide of claim 1, wherein the TALEN protein binds to a nucleotide sequence comprising one of SEQ ID NOs: 16-46, 50, 52, 58, and 59, or a complement thereof.

### Claim 3 (depends on 1)

The isolated polynucleotide of claim 1, wherein the TALEN protein comprises a nuclease.

### Claim 4 (depends on 3)

The isolated polynucleotide of claim 3, wherein the nuclease comprises FokI.

### Claim 5 (depends on 1)

The isolated polynucleotide of claim 1, wherein the TALEN protein comprises 15-19 repeat variable diresidue (RVD) modules.

### Claim 6 (depends on 1)

A vector comprising the isolated polynucleotide of claim 1.

### Claim 7 (depends on 6)

The vector of claim 6, wherein the TALEN protein binds to a nucleotide sequence comprising one of SEQ ID NOs: 16-46, 50, 52, 58, and 59, or a complement thereof.

### Claim 8 (depends on 6)

The vector of claim 6, wherein the TALEN protein comprises a nuclease.

### Claim 9 (depends on 8)

The vector of claim 8, wherein the nuclease comprises FokI.

### Claim 10 (depends on 6)

The vector of claim 6, wherein the TALEN protein comprises 15-19 repeat variable diresidue (RVD) modules.

### Claim 11 (depends on 1)

A cell comprising the isolated polynucleotide of claim 1.

### Claim 12 (depends on 11)

The cell of claim 11, wherein the TALEN protein binds to a nucleotide sequence comprising one of SEQ ID NOs: 16-46, 50, 52, 58, and 59, or a complement thereof.

### Claim 13 (depends on 11)

The cell of claim 11, wherein the TALEN protein comprises a nuclease.

### Claim 14 (depends on 13)

The cell of claim 13, wherein the nuclease comprises FokI.

### Claim 15 (depends on 11)

The cell of claim 11, wherein the TALEN protein comprises 15-19 repeat variable diresidue (RVD) modules.