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SG11201600060VA - Orthogonal cas9 proteins for rna-guided gene regulation and editing - Google Patents

Orthogonal cas9 proteins for rna-guided gene regulation and editing

Info

Publication number
SG11201600060VA
SG11201600060VA SG11201600060VA SG11201600060VA SG11201600060VA SG 11201600060V A SG11201600060V A SG 11201600060VA SG 11201600060V A SG11201600060V A SG 11201600060VA SG 11201600060V A SG11201600060V A SG 11201600060VA SG 11201600060V A SG11201600060V A SG 11201600060VA
Authority
SG
Singapore
Prior art keywords
editing
rna
gene regulation
cas9 proteins
guided gene
Prior art date
Application number
SG11201600060VA
Inventor
George M Church
Kevin Esvelt
Prashant Mali
Original Assignee
Harvard College
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Harvard College filed Critical Harvard College
Publication of SG11201600060VA publication Critical patent/SG11201600060VA/en

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SG11201600060VA 2013-07-10 2014-07-08 Orthogonal cas9 proteins for rna-guided gene regulation and editing SG11201600060VA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361844844P 2013-07-10 2013-07-10
PCT/US2014/045700 WO2015006294A2 (en) 2013-07-10 2014-07-08 Orthogonal cas9 proteins for rna-guided gene regulation and editing

Publications (1)

Publication Number Publication Date
SG11201600060VA true SG11201600060VA (en) 2016-02-26

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Application Number Title Priority Date Filing Date
SG11201600060VA SG11201600060VA (en) 2013-07-10 2014-07-08 Orthogonal cas9 proteins for rna-guided gene regulation and editing
SG10201800213VA SG10201800213VA (en) 2013-07-10 2014-07-08 Orthogonal cas9 proteins for rna-guided gene regulation and editing
SG10201913015XA SG10201913015XA (en) 2013-07-10 2014-07-08 Orthogonal cas9 proteins for rna-guided gene regulation and editing

Family Applications After (2)

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SG10201800213VA SG10201800213VA (en) 2013-07-10 2014-07-08 Orthogonal cas9 proteins for rna-guided gene regulation and editing
SG10201913015XA SG10201913015XA (en) 2013-07-10 2014-07-08 Orthogonal cas9 proteins for rna-guided gene regulation and editing

Country Status (12)

Country Link
US (4) US10329587B2 (en)
EP (2) EP3019204B1 (en)
JP (3) JP6718813B2 (en)
KR (2) KR102481330B1 (en)
CN (2) CN105517579B (en)
AU (4) AU2014287397B2 (en)
BR (1) BR112016000571B1 (en)
CA (2) CA3218040A1 (en)
IL (3) IL282489B (en)
RU (3) RU2704981C2 (en)
SG (3) SG11201600060VA (en)
WO (1) WO2015006294A2 (en)

Families Citing this family (123)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR085533A1 (en) 2011-03-23 2013-10-09 Pioneer Hi Bred Int PRODUCTION METHODS OF A COMPLEX TRANSGENIC RISK LOCUS
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
LT2800811T (en) 2012-05-25 2017-09-11 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP3138911B1 (en) 2012-12-06 2018-12-05 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
SG10201704932UA (en) 2012-12-17 2017-07-28 Harvard College Rna-guided human genome engineering
PL3456831T3 (en) 2013-04-16 2021-12-06 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
WO2015026887A1 (en) 2013-08-22 2015-02-26 E. I. Du Pont De Nemours And Company A soybean u6 polymerase iii promoter and methods of use
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150079680A1 (en) 2013-09-18 2015-03-19 Kymab Limited Methods, cells & organisms
WO2015070062A1 (en) * 2013-11-07 2015-05-14 Massachusetts Institute Of Technology Cell-based genomic recorded accumulative memory
US9834791B2 (en) 2013-11-07 2017-12-05 Editas Medicine, Inc. CRISPR-related methods and compositions with governing gRNAS
MX388127B (en) 2013-12-11 2025-03-19 Regeneron Pharma METHODS AND COMPOSITIONS FOR THE TARGETED MODIFICATION OF A GENOME.
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP4079847A1 (en) 2014-07-30 2022-10-26 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
RU2017112324A (en) 2014-09-12 2018-10-15 Пайонир Хай-Бред Интернэшнл, Инк. CREATION OF WEBSITES OF SITE-SPECIFIC INTEGRATION FOR COMPLEX SIGNS LOCUSES IN CORN AND SOY, AND ALSO WAYS OF APPLICATION
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
GB201418965D0 (en) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
US11680268B2 (en) 2014-11-07 2023-06-20 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
AU2015355546B2 (en) 2014-12-03 2021-10-14 Agilent Technologies, Inc. Guide RNA with chemical modifications
US9888673B2 (en) 2014-12-10 2018-02-13 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3274460A1 (en) 2015-03-27 2018-01-31 E. I. du Pont de Nemours and Company Soybean u6 small nuclear rna gene promoters and their use in constitutive expression of small rna genes in plants
EP3280803B1 (en) 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
EP3286571B1 (en) * 2015-04-24 2021-08-18 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
US11390884B2 (en) 2015-05-11 2022-07-19 Editas Medicine, Inc. Optimized CRISPR/cas9 systems and methods for gene editing in stem cells
WO2016186946A1 (en) * 2015-05-15 2016-11-24 Pioneer Hi-Bred International, Inc. Rapid characterization of cas endonuclease systems, pam sequences and guide rna elements
US11298410B2 (en) 2015-06-01 2022-04-12 Temple University—Of the Commonwealth System of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
KR102796744B1 (en) 2015-06-09 2025-04-15 에디타스 메디신, 인코포레이티드 CRISPR/CAS-related methods and compositions for improving transplantation
WO2016205745A2 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
WO2017023801A1 (en) 2015-07-31 2017-02-09 Regents Of The University Of Minnesota Intracellular genomic transplant and methods of therapy
AU2016326711B2 (en) 2015-09-24 2022-11-03 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/Cas-mediated genome editing
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
US20180305424A1 (en) * 2015-10-27 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Methods of Inducibly Targeting Chromatin Effectors and Compositions for Use in the Same
SG10202004557QA (en) * 2015-11-23 2020-06-29 Univ California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
BR112018013663A2 (en) 2016-01-11 2019-01-22 Univ Leland Stanford Junior chimeric proteins and immunotherapy methods
KR20180096800A (en) 2016-01-11 2018-08-29 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Methods of modulating chimeric proteins and gene expression
US11254928B2 (en) 2016-01-15 2022-02-22 Astrazeneca Ab Gene modification assays
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
WO2017180665A2 (en) 2016-04-12 2017-10-19 Edward Perkins Methods for creatings synthetic chromosomes expressing biosynthetic pathways and uses thereof
EP3910058B1 (en) * 2016-04-12 2025-01-08 CarryGenes Bioengineering, LLC Methods for creating synthetic chromosomes having gene regulatory systems and uses thereof
EP4047092A1 (en) 2016-04-13 2022-08-24 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
WO2017222834A1 (en) * 2016-06-10 2017-12-28 City Of Hope Compositions and methods for mitochondrial genome editing
BR112019001887A2 (en) 2016-08-02 2019-07-09 Editas Medicine Inc compositions and methods for treating cep290-associated disease
JP7231935B2 (en) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ Adenosine nucleobase editors and their uses
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
CN106282228A (en) * 2016-08-19 2017-01-04 苏州兰希亚生物科技有限公司 A kind of method that point mutation is repaired
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
EP3529359B1 (en) 2016-10-18 2023-12-13 Regents of the University of Minnesota Tumor infiltrating lymphocytes for use in therapy
EP3535396A1 (en) 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
EP3565907B1 (en) 2017-01-06 2022-05-04 Editas Medicine, Inc. Methods of assessing nuclease cleavage
AU2018212986B2 (en) * 2017-01-28 2024-07-25 Inari Agriculture Technology, Inc. Novel plant cells, plants, and seeds
TW201839136A (en) 2017-02-06 2018-11-01 瑞士商諾華公司 Composition and method for treating hemochromatosis
WO2018148647A2 (en) * 2017-02-10 2018-08-16 Lajoie Marc Joseph Genome editing reagents and their use
CA3054307A1 (en) * 2017-02-22 2018-08-30 Io Biosciences, Inc. Nucleic acid constructs comprising gene editing multi-sites and uses thereof
US10828330B2 (en) 2017-02-22 2020-11-10 IO Bioscience, Inc. Nucleic acid constructs comprising gene editing multi-sites and uses thereof
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
JP2020513814A (en) * 2017-03-14 2020-05-21 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Operation of CRISPR CAS9 Immune Stealth
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
JP7584777B2 (en) * 2017-03-30 2024-11-18 国立大学法人京都大学 Method for inducing exon skipping by genome editing
CN116555353A (en) 2017-04-20 2023-08-08 E开创生物技术股份有限公司 Method for producing genetically modified animals
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
CA3062595A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
KR102746733B1 (en) 2017-06-09 2024-12-24 에디타스 메디신, 인코포레이티드 Engineered CAS9 nuclease
JP2020530307A (en) 2017-06-30 2020-10-22 インティマ・バイオサイエンス,インコーポレーテッド Adeno-associated virus vector for gene therapy
EP3645721A1 (en) 2017-06-30 2020-05-06 Novartis AG Methods for the treatment of disease with gene editing systems
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
WO2019060746A1 (en) 2017-09-21 2019-03-28 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US11603536B2 (en) 2017-09-29 2023-03-14 Inari Agriculture Technology, Inc. Methods for efficient maize genome editing
AU2018352592B2 (en) 2017-10-16 2025-03-27 Beam Therapeutics, Inc. Uses of adenosine base editors
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
EP3704245A1 (en) 2017-11-01 2020-09-09 Novartis AG Synthetic rnas and methods of use
EP3707254A4 (en) * 2017-11-10 2021-08-18 University of Massachusetts PLATFORMS FOR TARGETED CRISPR RELEASE
US20200362366A1 (en) 2018-01-12 2020-11-19 Basf Se Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a
CA3091267A1 (en) 2018-02-23 2019-08-29 Pioneer Hi-Bred International, Inc. Novel cas9 orthologs
SG11202008956XA (en) 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
CN116349651A (en) 2018-03-19 2023-06-30 瑞泽恩制药公司 Transcriptional modulation in animals using the CRISPR/Cas system
JP6614622B2 (en) * 2018-04-17 2019-12-04 国立大学法人名古屋大学 Plant genome editing method
SG11202009783WA (en) 2018-04-19 2020-11-27 Univ California Compositions and methods for gene editing
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
GB201813011D0 (en) 2018-08-10 2018-09-26 Vib Vzw Means and methods for drought tolerance in crops
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
CN113166744B (en) 2018-12-14 2025-02-07 先锋国际良种公司 Novel CRISPR-Cas system for genome editing
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3696189A1 (en) * 2019-02-14 2020-08-19 European Molecular Biology Laboratory Means and methods for preparing engineered target proteins by genetic code expansion in a target protein selective manner
JP7669281B2 (en) 2019-03-19 2025-04-28 ザ ブロード インスティテュート,インコーポレーテッド Editing Methods and compositions for editing nucleotide sequences
US20210095273A1 (en) * 2019-09-30 2021-04-01 Sigma-Aldrich Co. Llc Modulation of microbiota compositions using targeted nucleases
EP4085133A1 (en) * 2019-12-30 2022-11-09 Lifeedit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
JP7525140B2 (en) * 2020-01-31 2024-07-30 国立大学法人京都大学 Protein translation control system
JP2023525304A (en) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
GB202007943D0 (en) * 2020-05-27 2020-07-08 Snipr Biome Aps Products & methods
RU2749307C1 (en) * 2020-10-30 2021-06-08 Федеральное государственное бюджетное научное учреждение "Всероссийский научно-исследовательский институт сельскохозяйственной биотехнологии" (ФГБНУ ВНИИСБ) New compact type ii cas9 nuclease from anoxybacillus flavithermus
WO2022116139A1 (en) * 2020-12-04 2022-06-09 中国科学院脑科学与智能技术卓越创新中心 Method for measuring levels of cellular endogenous low-abundance gene and lncrna
AU2022216614A1 (en) 2021-02-05 2023-02-23 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2023039586A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
IL312452A (en) 2021-11-01 2024-06-01 Tome Biosciences Inc Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
US20250057980A1 (en) 2021-12-22 2025-02-20 Tome Biosciences, Inc. Co-Delivery of a Gene Editor Construct and a Donor Template
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6933378B2 (en) * 1997-05-30 2005-08-23 Joseph Atabekov Methods for coexpression of more than one gene in eukaryotic cells
US6203986B1 (en) 1998-10-22 2001-03-20 Robert H. Singer Visualization of RNA in living cells
RU2467069C2 (en) * 2006-03-09 2012-11-20 Зе Скрипс Ресеч Инститьют System for expression of orthogonal translation components in eubacterial host cell
US8481272B2 (en) * 2006-08-04 2013-07-09 Georgia State University Research Foundation, Inc. Enzyme sensors, methods for preparing and using such sensors, and methods of detecting protease activity
MX2009009071A (en) 2007-03-02 2009-09-24 Danisco Cultures with improved phage resistance.
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
WO2010054108A2 (en) 2008-11-06 2010-05-14 University Of Georgia Research Foundation, Inc. Cas6 polypeptides and methods of use
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
MX2012013037A (en) 2010-05-10 2013-07-29 Univ California Endoribonuclease compositions and methods of use thereof.
EP3156062A1 (en) 2010-05-17 2017-04-19 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
CA2853829C (en) * 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2013126794A1 (en) 2012-02-24 2013-08-29 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
NZ629427A (en) 2012-02-29 2016-04-29 Sangamo Biosciences Inc Methods and compositions for treating huntington’s disease
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
LT2800811T (en) * 2012-05-25 2017-09-11 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014022702A2 (en) 2012-08-03 2014-02-06 The Regents Of The University Of California Methods and compositions for controlling gene expression by rna processing
EP3138911B1 (en) * 2012-12-06 2018-12-05 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
ES2576126T3 (en) * 2012-12-12 2016-07-05 The Broad Institute, Inc. Modification by genetic technology and optimization of improved enzyme systems, methods and compositions for sequence manipulation
US20140179770A1 (en) * 2012-12-12 2014-06-26 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
SG10201704932UA (en) * 2012-12-17 2017-07-28 Harvard College Rna-guided human genome engineering
KR20230136697A (en) * 2013-06-05 2023-09-26 듀크 유니버시티 Rna-guided gene editing and gene regulation

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