IP Library Granted Patent US 9,260,723
Granted Patent B2
US 9,260,723 · App. 14/319,255 · Granted Feb 16, 2016

RNA-guided human genome engineering

Inventors: Prashant G. Mali (Somerville, MA); George M. Church (Brookline, MA); Luhan Yang (Somerville, MA)
Assignee: President and Fellows of Harvard College
C12N15/85C12N15/01C12N15/102C12N15/63C12N15/81C12N15/8201C12N15/87C12N15/907C12N2810/55
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Quick Facts
Patent No.
US 9,260,723
App. No.
14/319,255
Filed
Jun 30, 2014
Granted
Feb 16, 2016
Kind
B2
Art Unit
1642
USPC
435/441
Abstract

A method of altering a eukaryotic cell is provided including transfecting the eukaryotic cell with a nucleic acid encoding RNA complementary to genomic DNA of the eukaryotic cell, transfecting the eukaryotic cell with a nucleic acid encoding an enzyme that interacts with the RNA and cleaves the genomic DNA in a site specific manner, wherein the cell expresses the RNA and the enzyme, the RNA binds to complementary genomic DNA and the enzyme cleaves the genomic DNA in a site specific manner.

Claims (9)

1. A method of integrating foreign DNA into a genomic nucleic acid sequence of a eukaryotic cell comprising providing to the eukaryotic cell a single stranded DNA (ssDNA) donor sequence tethered to a guide RNA sequence via hybridization, wherein the guide RNA sequence comprises a spacer sequence complementary to a target nucleic acid sequence, providing to the eukaryotic cell a Cas9 enzyme that interacts with the guide RNA sequence and cleaves the target nucleic acid sequence in a site specific manner, wherein the spacer sequence of the guide RNA sequence binds to the complementary target nucleic acid sequence and the Cas9 enzyme cleaves the target nucleic acid sequence in a site specific manner; and wherein the ssDNA donor sequence is integrated into the genomic nucleic acid sequence.

2. The method of claim 1 wherein the ssDNA donor nucleic acid is integrated into the genomic nucleic acid sequence by homologous recombination.

3. The method of claim 1 wherein the guide RNA sequence is provided to the cell by introducing to the cell a nucleic acid encoding the guide RNA sequence, wherein the Cas9 enzyme is provided to the cell by introducing to the cell a nucleic acid encoding the Cas9 enzyme, and wherein the cell expresses the guide RNA and the Cas9 protein.

4. The method of claim 1 wherein the eukaryotic cell is a mammalian cell.

5. The method of claim 1 wherein the eukaryotic cell is a yeast cell, a plant cell or a mammalian cell.

6. The method or claim 1 wherein the eukaryotic cell is a human cell.

7. The method of claim 1 wherein the guide RNA sequence is between about 10 to about 250 nucleotides.

8. The method of claim 1 wherein the guide RNA sequence is between about 20 to about 100 nucleotides.

9. The method of claim 1 wherein the guide RNA sequence is between about 100 to about 250 nucleotides.

Assignments (2)
CONFIRMATORY LICENSE Recorded Nov 24, 2014
From: HARVARD UNIVERSITY
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 034432/0597 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 3, 2014
From: CHURCH, GEORGE M.; MALI, PRASHANT; YANG, LUHAN
To: PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Reel/Frame 034087/0912 →
Continuity (4)
Continuation PCTUS2013075317 · Dec 16, 2013
Provisional Application 61738355 · Dec 17, 2012
Provisional Application 61779169 · Mar 13, 2013
Related Publication 20140342458A1 · Nov 20, 2014