RNA-guided transcriptional regulation
Abstract
Methods of modulating expression of a target nucleic acid in a cell are provided including introducing into the cell a first foreign nucleic acid encoding one or more RNAs complementary to DNA, wherein the DNA includes the target nucleic acid, introducing into the cell a second foreign nucleic acid encoding a nuclease-null Cas9 protein that binds to the DNA and is guided by the one or more RNAs, introducing into the cell a third foreign nucleic acid encoding a transcriptional regulator protein or domain, wherein the one or more RNAs, the nuclease-null Cas9 protein, and the transcriptional regulator protein or domain are expressed, wherein the one or more RNAs, the nuclease-null Cas9 protein and the transcriptional regulator protein or domain co-localize to the DNA and wherein the transcriptional regulator protein or domain regulates expression of the target nucleic acid.
- Inventors:
- Issue Date:
- Research Org.:
- Harvard Univ., Cambridge, MA (United States)
- Sponsoring Org.:
- USDOE
- OSTI Identifier:
- 1735230
- Patent Number(s):
- 10767194
- Application Number:
- 16/441,209
- Assignee:
- President and Fellows of Harvard College (Cambridge, MA)
- Patent Classifications (CPCs):
-
C - CHEMISTRY C12 - BIOCHEMISTRY C12N - MICROORGANISMS OR ENZYMES
C - CHEMISTRY C12 - BIOCHEMISTRY C12Y - ENZYMES
- DOE Contract Number:
- FG02-02ER63445
- Resource Type:
- Patent
- Resource Relation:
- Patent File Date: 06/14/2019
- Country of Publication:
- United States
- Language:
- English
- Subject:
- 59 BASIC BIOLOGICAL SCIENCES
Citation Formats
Church, George M., Mali, Prashant G., and Esvelt, Kevin M. RNA-guided transcriptional regulation. United States: N. p., 2020.
Web.
Church, George M., Mali, Prashant G., & Esvelt, Kevin M. RNA-guided transcriptional regulation. United States.
Church, George M., Mali, Prashant G., and Esvelt, Kevin M. Tue .
"RNA-guided transcriptional regulation". United States. https://www.osti.gov/servlets/purl/1735230.
@article{osti_1735230,
title = {RNA-guided transcriptional regulation},
author = {Church, George M. and Mali, Prashant G. and Esvelt, Kevin M.},
abstractNote = {Methods of modulating expression of a target nucleic acid in a cell are provided including introducing into the cell a first foreign nucleic acid encoding one or more RNAs complementary to DNA, wherein the DNA includes the target nucleic acid, introducing into the cell a second foreign nucleic acid encoding a nuclease-null Cas9 protein that binds to the DNA and is guided by the one or more RNAs, introducing into the cell a third foreign nucleic acid encoding a transcriptional regulator protein or domain, wherein the one or more RNAs, the nuclease-null Cas9 protein, and the transcriptional regulator protein or domain are expressed, wherein the one or more RNAs, the nuclease-null Cas9 protein and the transcriptional regulator protein or domain co-localize to the DNA and wherein the transcriptional regulator protein or domain regulates expression of the target nucleic acid.},
doi = {},
journal = {},
number = ,
volume = ,
place = {United States},
year = {Tue Sep 08 00:00:00 EDT 2020},
month = {Tue Sep 08 00:00:00 EDT 2020}
}
Works referenced in this record:
Cell-Penetrating Peptide-Mediated Delivery of TALEN Proteins via Bioconjugation for Genome Engineering
journal, January 2014
- Liu, Jia; Gaj, Thomas; Patterson, James T.
- PLoS ONE, Vol. 9, Issue 1
Diverse CRISPRs Evolving in Human Microbiomes
journal, June 2012
- Rho, Mina; Wu, Yu-Wei; Tang, Haixu
- PLoS Genetics, Vol. 8, Issue 6, Article No. e1002441
Prokaryotic RNAi-Like System and Methods of Use
patent-application, August 2011
- Terns, Rebecca; Terns, Michael; Hale, Caryn
- US Patent Application 13/055769; 20110189776
The VP16 transcription activation domain is functional when targeted to a promoter-proximal RNA sequence.
journal, November 1992
- Tiley, L. S.; Madore, S. J.; Malim, M. H.
- Genes & Development, Vol. 6, Issue 11
Target DNA interference with crRNA
patent-application, March 2010
- Sontheimer, Erik J.; Marraffini, Luciano A.
- US Patent Application 12/565589; 20100076057
Essential Features and Rational Design of CRISPR RNAs that Function with the Cas RAMP Module Complex to Cleave RNAs
journal, February 2012
- Hale, Caryn R.; Majumdar, Sonali; Elmore, Joshua
- Molecular Cell, Vol. 45, Issue 3, p. 292-302
Robust, synergistic regulation of human gene expression using TALE activators
journal, February 2013
- Maeder, Morgan L.; Linder, Samantha J.; Reyon, Deepak
- Nature Methods, Vol. 10, Issue 3
Crisper-Cas Nickase Systems, Methods and Compositions for Sequence Manipulation in Eukaryotes
patent-application, October 2014
- Zhang, Feng; Cong, Le; Ran, Fei
- US Patent Application 14/105031; 20140310830
CRISPR-Cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
patent, January 2015
- Cong, Le; Zhang, Feng
- US Patent Document 8,932,814
Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
patent-application, June 2014
- Zhang, Feng; Ran, Fei
- US Patent Application 14/104837; 20140179770
Synergistic and tunable human gene activation by combinations of synthetic transcription factors
journal, February 2013
- Perez-Pinera, Pablo; Ousterout, David G.; Brunger, Jonathan M.
- Nature Methods, Vol. 10, Issue 3
CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
journal, July 2013
- Gilbert, Luke A.; Larson, Matthew H.; Morsut, Leonardo
- Cell, Vol. 154, Issue 2, p. 442-451
Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression
journal, February 2013
- Qi, Lei S.; Larson, Matthew H.; Gilbert, Luke A.
- Cell, Vol. 152, Issue 5, p. 1173-1183
Methods And Compositions For Rna-Directed Target Dna Modification And For Rna-Directed Modulation Of Transcription
patent-application, March 2014
- Doudna, Jennifer A.; Jinek, Martin; Charpentier, Emmanuelle
- US Patent Application 13/842859; 20140068797
CRISPR-Cas Component Systems, Methods and Compositions for Sequence Manipulation
patent-application, June 2014
- Zhang, Feng
- US Patent Application 14/183486; 20140179006
Increasing Specificity for RNA-Guided Genome Editing
patent-application, January 2016
- Joung, J. Keith; Angstman, James; Sander, Jeffry D.
- US Patent Application 14/776620; 20160024524
Engineering of Systems, Methods, and Optimized Guide Compositions with New Architectures for Sequence Manipulation
patent-application, December 2016
- Ran, Fei; Zhang, Fend
- US Patent Application 15/172636; 20160355795
A CRISPR Approach to Gene Targeting
journal, September 2012
- Carroll, Dana
- Molecular Therapy, Vol. 20, Issue 9, p. 1658-1660
The Replication Fork: Understanding the Eukaryotic Replication Machinery and the Challenges to Genome Duplication
journal, January 2013
- Leman, Adam; Noguchi, Eishi
- Genes, Vol. 4, Issue 1
Crispr-based genome modification and regulation
patent-application, September 2014
- Chen, Fuqiang; Davis, Gregory
- US Patent Application 14/292212; 20140273233
RNA-Guided Human Genome Engineering via Cas9
journal, January 2013
- Mali, P.; Yang, L.; Esvelt, K. M.
- Science, Vol. 339, Issue 6121, p. 823-826
Riboactivators: Transcription activation by noncoding RNA
journal, February 2009
- Ansari, Aseem Z.
- Critical Reviews in Biochemistry and Molecular Biology, Vol. 44, Issue 1
CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering
journal, August 2013
- Mali, Prashant; Aach, John; Stranges, P. Benjamin
- Nature Biotechnology, Vol. 31, Issue 9
Multiplex Genome Engineering Using CRISPR/Cas Systems
journal, January 2013
- Cong, L.; Ran, F. A.; Cox, D.
- Science, Vol. 339, Issue 6121, p. 819-823
RNA-Guided Human Genome Engineering
patent-application, November 2014
- Mali, Prashant G.; Church, George M.; Yang, Luhan
- US Patent Application 14/319171; 20140342457
Engineering of systems, methods and optimized guide compositions for sequence manipulation
patent-application, September 2015
- Zhang, Feng; Cong, Le; Hsu, Patrick
- US Patent Application 14/704551; 20150247150
The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli
journal, August 2011
- Sapranauskas, Rimantas; Gasiunas, Giedrius; Fremaux, Christophe
- Nucleic Acids Research, Vol. 39, Issue 21
Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system
journal, August 2013
- Cheng, Albert W.; Wang, Haoyi; Yang, Hui
- Cell Research, Vol. 23, Issue 10
Systems and methods for reading data from a storage medium
patent, February 2016
- Ong, Chun Lian; Yuan, Zhimin; Leong, Siang Huei
- US Patent Document 9,257,135
Methods of Generating Nucleic Acid Fragments
patent-application, September 2011
- Wiedenheft, Blake; Zhou, Kaihong; Doudna, Jennifer A.
- US Patent Application 13/039160; 20110223638
Endroibonuclease Compositions and Methods of Use Thereof
patent-application, May 2013
- Haurwitz, Rachel E.; Doudna, Jennifer A.; Wiedenheft, Blake
- US Patent Application 13/671120; 2013/0130248
Optimized Crisper-Cas Double Nickase Systems, Methods and Compositions for Sequence Manipulation
patent-application, June 2016
- Zhang, Feng; Hsu, Patrick; Lin, Chie-yu
- US Patent Application 14/972523; 20160153006
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
journal, June 2012
- Jinek, M.; Chylinski, K.; Fonfara, I.
- Science, Vol. 337, Issue 6096, p. 816-821
Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
patent, April 2015
- Zhang, Feng; Cong, Le; Platt, Randall Jeffrey
- US Patent Document 8,999,641
Compositions and methods of nucleic acid-targeting nucleic acids
patent-application, October 2014
- May, Andrew Paul; Haurwitz, Rachel E.; Doudna, Jennifer A.
- US Patent Application 14/206319; 20140315985
Engineering of systems, methods and optimized guide compositions for sequence manipulation
patent, December 2014
- Zhang, Feng; Cong, Le; Hsu, Patrick D.
- US Patent Document 8,906,616
Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria
journal, September 2012
- Gasiunas, G.; Barrangou, R.; Horvath, P.
- Proceedings of the National Academy of Sciences, Vol. 109, Issue 39, p. E2579-E2586
CRISPR/CAS Systems for Genomic Modification and Gene Modulation
patent-application, September 2014
- Wu, Fangting
- US Patent Application 14/216655; 20140273226
RNA-guided genetic silencing systems in bacteria and archaea
journal, February 2012
- Wiedenheft, Blake; Sternberg, Samuel H.; Doudna, Jennifer A.
- Nature, Vol. 482, Issue 7385, p. 331-338
Engineering of Systems, Methods and Optimized Guide Compositions for Sequence Manipulation
patent-application, November 2016
- Zhang, Feng; Cong, Le; Hsu, Patrick
- US Patent Application 15/230025; 2016/0340662
Evolution and classification of the CRISPR–Cas systems
journal, May 2011
- Makarova, Kira S.; Haft, Daniel H.; Barrangou, Rodolphe
- Nature Reviews Microbiology, Vol. 9, Issue 6, p. 467-477
CRISPR-Cas systems and methods for altering expression of gene products
patent, April 2014
- Zhang, Feng
- US Patent Document 8,697,359
Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
patent, March 2015
- Zhang, Feng; Cong, Le; Platt, Randall Jeffrey
- US Patent Document 8,993,233
Mature clustered, regularly interspaced, short palindromic repeats RNA (crRNA) length is measured by a ruler mechanism anchored at the precursor processing site
journal, December 2011
- Hatoum-Aslan, A.; Maniv, I.; Marraffini, L. A.
- Proceedings of the National Academy of Sciences, Vol. 108, Issue 52, p. 21218-21222
Methods and compositions for treating Huntington's disease
patent-application, September 2013
- Miller, Jeffrey C.; Rebar, Edward J.; Zhang, H. Steve
- US Patent Application 13/781176; 20130253040
RNA-Guided Human Genome Engineering
patent-application, June 2016
- Mali, Prashant G.; Church, George M.; Yang, Luhan
- US Patent Application 15/042515; 20160160210
Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA
journal, April 2014
- Ramakrishna, S.; Kwaku Dad, A. -B.; Beloor, J.
- Genome Research, Vol. 24, Issue 6
Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity
journal, September 2013
- Ran, F. Ann; Hsu, Patrick D.; Lin, Chie-Yu
- Cell, Vol. 154, Issue 6, p. 1380-1389
RNA-guided transcriptional regulation
patent, February 2016
- Church, George M.; Mali, Prashant G.; Esvelt, Kevin M.
- US Patent Document 9,267,135
Clustered regularly interspaced short palindromic repeats (CRISPRs): the hallmark of an ingenious antiviral defense mechanism in prokaryotes
journal, July 2011
- Al-Attar, Sinan; Westra, Edze R.; van der Oost, John
- Biological Chemistry, Vol. 392, Issue 4, p. 277-289
CRISPR-Cas component systems, methods and compositions for sequence manipulation
patent, October 2014
- Cong, Le; Zhang, Feng
- US Patent Document 8,871,445
Crisper-Cas Nickase Systems, Methods and Compostions for Sequence Manipulations in Eukaryotes
patent-application, July 2017
- Zhang, Feng; Cong, Le; Ran, Fei
- US Patent Application 15/229702; 20170191078