IP Library Granted Patent US 9,493,750
Granted Patent B2
US 9,493,750 · App. 14/065,028 · Granted Nov 15, 2016

DNA-binding proteins and uses thereof

Inventors: Philip D. Gregory (Richmond, CA); Jeffrey C. Miller (Richmond, CA); David Paschon (Richmond, CA); Edward J. Rebar (Richmond, CA); Siyuan Tan (Richmond, CA); Fyodor Urnov (Richmond, CA); Lei Zhang (Richmond, CA)
Assignee: Sangamo BioSciences, Inc.
C12N9/22C12N15/52C12N15/62C12N15/85A61K38/00C07K2319/80
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Quick Facts
Patent No.
US 9,493,750
App. No.
14/065,028
Granted
Nov 15, 2016
Kind
B2
Abstract

Disclosed herein are polypeptides, polynucleotides encoding, cells and organisms comprising novel DNA-binding domains, including TALE DNA-binding domains. Also disclosed are methods of using these novel DNA-binding domains for modulation of gene expression and/or genomic editing of endogenous cellular sequences.

Claims (15)

1. An isolated, non-naturally occurring polynucleotide encoding a non-naturally occurring fusion protein comprising:

(i) at least one functional domain, wherein the functional domain comprises a transcriptional activator, a transcriptional repressor or a nuclease; and

(ii) a non-naturally occurring DNA-binding polypeptide comprising:

two or more TALE-repeat units, the TALE repeat units comprising a repeat variable di-residue (RVD), wherein at least one TALE-repeat unit is non-naturally occurring;

an N-cap polypeptide, wherein the N-cap comprises a fragment of a full length N-terminus region of a TALE protein and does not comprise a full length N-terminal region of a TALE protein, the N-cap comprising residues N+1 to N+121 of a TALE protein; and

a C-cap polypeptide, wherein the C-cap polypeptide comprises a fragment of a full length C-terminus region of a TA LE protein and does not comprise a full length C-terminal region of a TALE protein, the C-cap comprising residues C-1 to C-20 of the TALE protein, wherein the polypeptide binds to DNA.

2. The polynucleotide of claim 1 , wherein at least one TALE-repeat unit comprises art atypical repeat variable di-residue (RVD).

3. The polynucleotide of claim 2 , wherein the protein comprises an atypical RVD as shown in Tables 27A or 27B.

4. The polynucleotide of claim 1 , wherein the C-cap polypeptide is less than approximately 230 amino acids in length.

5. The polynucleotide of claim 1 , wherein the C-cap comprises a TALE repeat domain.

6. The polynucleotide of claim 1 , wherein the functional domain is a transcriptional activator or a transcriptional repressor.

7. The polynucleotide of claim 1 , wherein the functional domain comprises a nuclease.

8. The polynucleotide of claim 7 , wherein the nuclease comprises at least one cleavage domain or cleavage half-domain from a TypeIIS endonuclease.

9. An isolated host cell comprising a polynucleotide according to claim 1 .

10. A pharmaceutical composition comprising a polynucleotide according to claim 1 .

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 29, 2022
From: GREGORY, PHILIP D.; MILLER, JEFFREY C.; PASCHON, DAVID; REBAR, EDWARD J.; TAN, SIYUAN; URNOV, FYODOR; ZHANG, LEI
To: SANGAMO BIOSCIENCES, INC.
Reel/Frame 060674/0666 →
CHANGE OF NAME Recorded Jul 12, 2017
From: SANGAMO BIOSCIENCES, INC.
To: SANGAMO THERAPEUTICS, INC.
Reel/Frame 043177/0014 →
Continuity (8)
Division 13068735 · May 17, 2011
Provisional Application 61395836 · May 17, 2010
Provisional Application 61401429 · Aug 12, 2010
Provisional Application 61455121 · Oct 13, 2010
Provisional Application 61459891 · Dec 20, 2010
Provisional Application 61462482 · Feb 2, 2011
Provisional Application 61465869 · Mar 24, 2011
Related Publication 20140134723A1 · May 15, 2014