CRISPR-Cas effector polypeptides and methods of use thereof
AssigneeThe Regents of the University of California
InventorsJennifer A. Doudna, Basem Al-Shayeb, Jillian F. Banfield, Patrick Pausch
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Abstract
The present disclosure provides RNA-guided CRISPR-Cas effector proteins, nucleic acids encoding same, and compositions comprising same. The present disclosure provides ribonucleoprotein complexes comprising: an RNA-guided CRISPR-Cas effector protein of the present disclosure; and a guide RNA. The present disclosure provides methods of modifying a target nucleic acid, using an RNA-guided CRISPR-Cas effector protein of the present disclosure and a guide RNA. The present disclosure provides methods of modulating transcription of a target nucleic acid.
Claims38
A pharmaceutical composition comprising:
- a) at least one expression vector encoding:
- i) a polypeptide comprising an amino acid sequence that is at least 95% identical to SEQ ID NO:
- 120;
- and
- ii) a recombinant guide RNA comprising:
- 1) A constant region comprising a first nucleotide sequence that is at least 80% identical to SEQ ID NO:
- 181;
- and
- 2) a targeting segment comprising a second nucleotide sequence
- complementary to a target sequence on a target strand (TS) of a double-stranded DNA molecule, wherein the double-stranded DNA molecule comprises a 5′-NTTN-3′ protospacer adjacent motif (PAM) sequence positioned immediately 5′ of the target sequence on a non-target strand (NTS) of the double stranded DNA molecule, wherein T is thymine and N is any nucleotide, and
- wherein a 3′ end of the first nucleotide sequence of the recombinant guide RNA is linked to a 5′ end of the second nucleotide sequence;
- and
- b) a pharmaceutically acceptable excipient.
Dependencies are inferred from the claim text.