Novel rna-programmable endonuclease systems and uses thereof
AssigneesCrispr Therapeutics AG, Bayer HealthCare LLC
InventorsAndre COHNEN, Moritz SCHMIDT, Wayne COCO, Michael Biag GAMALINDA, Ashish GUPTA, Christian PITZLER, Florian RICHTER, Jan TEBBE, Christopher CHENG, Ryo TAKEUCHI, Caroline W. REISS
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Abstract
Aspects of this invention, inter alia, relate to novel systems for targeting, editing or manipulating DNA in a cell, using novel synthetic RNA-guided nucleases (sRGNs). The sRGNs are derived from wildtype or parental small type II CRISPR Cas9 endonucleases.
Claims11
1-41. (canceled)
A synthetic RNA-guided nuclease (sRGN) polypeptide comprising an amino acid sequence having at least 95% identity to the amino acid sequence of SEQ ID NO:
- 3 or 4.
The sRGN polypeptide of SEQ ID NO:
- 3 or 4, wherein the amino acid sequence has at least 96%, 97%, 98%, or 99% identity to the amino acid sequence of SEQ ID NO:
- 3 or 4, or wherein the amino acid sequence is SEQ ID NO:
- 3 or 4.
A system for introducing a single-stranded or a double-stranded break in a target polynucleotide, the system comprising:
- (a) a sRGN polypeptide comprising an amino acid sequence having at least 95% identity to the amino acid sequence of SEQ ID NO:
- 3 or 4;
- and
- (b) a single guide RNA (sgRNA) comprising a DNA targeting segment capable of hybridizing to a target polynucleotide,
- wherein the sgRNA combines with the sRGN polypeptide to induce a single-stranded or double-stranded break in the target polynucleotide.
Dependencies are inferred from the claim text.