Title:
DIRECTED EDITING OF CELLULAR RNA VIA NUCLEAR DELIVERY OF CRISPR/CAS9
Document Type and Number:
Japanese Patent JP2023106391
Kind Code:
A
Abstract:
To provide a technology to perform programmable RNA editing at single-nucleotide resolution using RNA-targeting CRISPR/Cas9.SOLUTION: A recombinant expression system for CRISPR/Cas-directed RNA editing of a target RNA comprises: (A) a nucleic acid sequence encoding a CRISPR/Cas RNA editing fusion protein comprising a nuclease-dead CRISPR associated endonuclease (dCas) fused to a catalytically active deaminase domain of Adenosine Deaminase acting on RNA (ADAR); and (B) a nucleic acid sequence encoding an extended single guide RNA (esgRNA) comprising: (i) a short extension sequence of homology to the target RNA comprising a mismatch for a target adenosine, and (ii) a dCas scaffold binding sequence.SELECTED DRAWING: None
Inventors:
YEO EUGENE
KRISTOPHER BRANNAN
RYAN MARINA
DAVID NELLES
KRISTOPHER BRANNAN
RYAN MARINA
DAVID NELLES
Application Number:
JP2023070698A
Publication Date:
August 01, 2023
Filing Date:
April 24, 2023
Export Citation:
Assignee:
UNIV CALIFORNIA
International Classes:
C12N15/09; A61K35/12; A61K35/76; A61K35/761; A61K48/00; A61P21/00; A61P25/00; A61P25/14; A61P25/16; A61P25/28; A61P25/32; A61P27/16; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N7/01; C12N15/11
Attorney, Agent or Firm:
Hiroshi Kobayashi
Norio Omori
Norio Omori
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