Title:
ENGINEERED CRISPR-CAS9 COMPOSITIONS AND METHODS OF USE
Document Type and Number:
WIPO Patent Application WO/2017/027423
Kind Code:
A9
Abstract:
The present specification discloses engineered Type II CRISPR-Cas9-associated split-nexus polynucleotide (sn-casPNs) systems, including systems comprising three split- nexus polynucleotides (sn1-casPN/sn2-casPN/sn3-casPN) and systems comprising two split-nexus polynucleotides (sn1-casPN/sn2-casPN). Together with a Cas9 protein, the sn-casPNs facilitate site-specific modifications, including cleavage and mutagenesis, of a target polynucleotide in
vitro and in
vivo. Furthermore, the engineered Type II CRISPR-Cas9-associated split-nexus polynucleotide systems comprising sn-casPNs are useful in methods of regulating expression of a target nucleic acid. Methods are described herein for the creation of a variety of engineered Type II CRISPR-Cas9-associated split-nexus polynucleotide systems comprising two or more sn-casPNs. Polynucleotide sequences, expression cassettes, vectors, compositions, and kits for carrying out a variety of methods are also described. Furthermore, the present specification provides genetically modified cells, compositions of modified cells, transgenic organisms, pharmaceutical compositions, as well as a variety of compositions and methods involving the engineered Type II CRISPR-Cas9-associated split-nexus polynucleotide systems.
Inventors:
DONOHOUE PAUL (US)
MAY ANDREW (US)
MAY ANDREW (US)
Application Number:
PCT/US2016/045915
Publication Date:
July 05, 2018
Filing Date:
August 05, 2016
Export Citation:
Assignee:
CARIBOU BIOSCIENCES INC (US)
International Classes:
C12N15/11; C12N15/09; C12N15/63
Attorney, Agent or Firm:
MCCLUNG, Barbara et al. (US)
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