IP Library Granted Patent US 10,494,649
Granted Patent B2
US 10,494,649 · App. 15/980,450 · Granted Dec 3, 2019

Systems and methods for one-shot guide RNA (ogRNA) targeting of endogenous and source DNA

Inventors: Ari E. Friedland (Boston, MA); Hariharan Jayaram (Cambridge, MA); Barrett Ethan Steinberg (Cambridge, MA)
Assignee: EDITAS MEDICINE, INC.
C12N15/907C12N9/22C12N9/96C12N15/102C12N15/11C12N15/113C12N15/63C12N2310/20C12N2810/10
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Quick Facts
Patent No.
US 10,494,649
App. No.
15/980,450
Granted
Dec 3, 2019
Kind
B2
Abstract

Engineered nucleic acids encoding genome editing system components are provided, as are engineered RNA-guided nucleases that include inserts encoded in part by cellular genomic or other sequences recognized by guide RNAs.

Claims (13)

1. An engineered RNA-guided nuclease protein comprising an amino acid insertion or substitution at least partially encoded by a eukaryotic nucleic acid sequence of at least 17 nucleotides in length,

wherein the engineered RNA-guided nuclease is a Cas9 protein,

wherein the Cas9 protein comprises an amino acid insertion relative to SEQ ID NO: 2 selected from the group consisting of:

E271_N272insGX 6-10 G,

L371_N372insGX 6-10 G, and

Q737_A738insGX 6-10 G; or

wherein the Cas9 protein comprises an amino acid insertion S12_M13insGX 6-10 G relative to SEQ ID NO: 10,

wherein the insertion relative to SEQ ID NO: 2 or SEQ ID NO: 10 results in a polypeptide comprising an amino acid sequence having at least 95% sequence identity to a sequence selected from the group consisting of SEQ ID NOs: 3-5 and 10 and having an RNA-guided nuclease activity.

2. The RNA-guided nuclease protein of claim 1 , wherein the RNA-guided nuclease has at least about 80% nuclease activity of a wild-type RNA-guided nuclease.

3. The RNA-guided nuclease protein of claim 1 , wherein the eukaryotic nucleic acid sequence is a mammalian sequence.

4. The RNA-guided nuclease protein of claim 1 , wherein the eukaryotic nucleic acid sequence comprises or is adjacent to a protospacer adjacent motif (PAM) that is recognized by the engineered RNA-guided nuclease protein.

5. The RNA-guided nuclease protein of claim 4 , wherein the eukaryotic nucleic acid sequence comprises at least 17 nucleotides adjacent to the PAM.

6. The RNA-guided nuclease protein of claim 1 , wherein the Cas9 protein further comprises an insertion at or near the N-terminus of the Cas9 protein.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 11, 2020
From: FRIEDLAND, ARI E.; JAYARAM, HARIHARAN; STEINBERG, BARRETT ETHAN
To: EDITAS MEDICINE, INC.
Reel/Frame 051787/0550 →
Continuity (5)
Division 15934750 · Mar 23, 2018
Division 15832567 · Dec 5, 2017
Provisional Application 62503640 · May 9, 2017
Provisional Application 62430154 · Dec 5, 2016
Related Publication 20180251792A1 · Sep 6, 2018