US2022356493A1PendingUtilityA1

Dna-binding proteins and uses thereof

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Assignee: SANGAMO THERAPEUTICS INCPriority: May 17, 2010Filed: Jun 11, 2021Published: Nov 10, 2022
Est. expiryMay 17, 2030(~3.8 yrs left)· nominal 20-yr term from priority
Inventors:Philip D. GregoryJeffrey C. MillerDavid PaschonEdward J. RebarSiyuan TanFyodor UrnovLei Zhang
C12Y 301/21004C07K 19/00C12N 15/8213C12N 15/52A61P 27/02A61K 38/16C12N 15/62A61K 48/00C12N 9/22A61P 43/00A61K 38/00C12N 15/85C07K 2319/80C07K 14/195C12N 15/00C12N 15/907A61P 25/00
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Claims

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

exact text as granted — not AI-modified
What is claims is: 
     
         1 . An isolated polynucleotide encoding a non-naturally occurring TALE DNA-binding polypeptide comprising:
 two or more TALE-repeat units, the TALE repeat units comprising a repeat variable di-residue (RVD); and wherein the N-terminal region lacks at least 152 amino acids of a full-length TALE protein and the C-terminal region comprises fragment of a full-length C-terminal TALE protein, and further wherein the polypeptide binds to DNA.   
     
     
         2 . The polynucleotide of  claim 1 , wherein at least one TALE-repeat unit comprises an atypical repeat variable di-residue (RVD). 
     
     
         3 . The polynucleotide of  claim 2 , wherein the polypeptide comprises an atypical RVD as shown in Tables 27A or 27B. 
     
     
         4 . The polynucleotide of  claim 1 , wherein the N-terminal region of the polypeptide is truncated to residue N+134 to residue N+137. 
     
     
         5 . The polynucleotide of  claim 1 , wherein the C-cap of the polypeptide comprises a TALE repeat domain. 
     
     
         6 . The polynucleotide of  claim 1 , wherein the polypeptide further comprises a functional domain. 
     
     
         7 . The polynucleotide of  claim 6 , wherein the functional domain is a transcriptional activator or a transcriptional repressor. 
     
     
         8 . The polynucleotide of  claim 1 , wherein the functional domain comprises a nuclease domain. 
     
     
         9 . The polynucleotide of  claim 8 , wherein the nuclease domain is an endonuclease domain. 
     
     
         10 . A cell comprising the polynucleotide of  claim 1 . 
     
     
         11 . A pharmaceutical composition comprising the polynucleotide of  claim 1 . 
     
     
         12 . A pharmaceutical composition comprising a cell according to  claim 10 . 
     
     
         13 . An isolated polynucleotide encoding a non-naturally occurring TALE DNA-binding polypeptide comprising:
 two or more TALE-repeat units, the TALE repeat units comprising a repeat variable di-residue (RVD); and wherein the N-terminal region comprises amino acids N+1 to N+121 of a full-length TALE protein and the C-terminal region comprises no more than C−20 to C+63 of a full length TALE protein, and further wherein the polypeptide binds to DNA.   
     
     
         14 . The polynucleotide of  claim 12 , wherein at least one TALE-repeat unit comprises an atypical repeat variable di-residue (RVD). 
     
     
         15 . The polynucleotide of  claim 14 , wherein the polypeptide comprises an atypical RVD as shown in Tables 27A or 27B. 
     
     
         16 . The polynucleotide of  claim 13 , wherein the N-terminal region of the polypeptide is truncated to residue N+134 to residue N+137. 
     
     
         17 . The polynucleotide of  claim 13 , wherein the C-cap of the polypeptide comprises a TALE repeat domain. 
     
     
         18 . The polynucleotide of  claim 13 , wherein the polypeptide further comprises a functional domain. 
     
     
         19 . The polynucleotide of  claim 18 , wherein the functional domain is a transcriptional activator or a transcriptional repressor. 
     
     
         20 . The polynucleotide of  claim 13 , wherein the functional domain comprises a nuclease domain. 
     
     
         21 . The polynucleotide of  claim 20 , wherein the nuclease domain is an endonuclease domain. 
     
     
         22 . A cell comprising the polynucleotide of  claim 13 . 
     
     
         23 . A pharmaceutical composition comprising the polynucleotide of  claim 13 . 
     
     
         24 . A pharmaceutical composition comprising a cell according to  claim 22 .

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