EP4274894A2 - Prime-editor-varianten, konstrukte und verfahren zur verbesserung der prime-editierungseffizienz und -präzision - Google Patents

Prime-editor-varianten, konstrukte und verfahren zur verbesserung der prime-editierungseffizienz und -präzision

Info

Publication number
EP4274894A2
EP4274894A2 EP22702103.7A EP22702103A EP4274894A2 EP 4274894 A2 EP4274894 A2 EP 4274894A2 EP 22702103 A EP22702103 A EP 22702103A EP 4274894 A2 EP4274894 A2 EP 4274894A2
Authority
EP
European Patent Office
Prior art keywords
fold
seq
dna
amino acid
basepair
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
EP22702103.7A
Other languages
English (en)
French (fr)
Inventor
David R. Liu
Peter J. CHEN
Brittany ADAMSON
Jeffrey HUSSMANN
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Harvard College
University of California
Princeton University
Broad Institute Inc
Original Assignee
Harvard College
University of California
Princeton University
Broad Institute Inc
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, University of California, Princeton University, Broad Institute Inc filed Critical Harvard College
Publication of EP4274894A2 publication Critical patent/EP4274894A2/de
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241Nucleotidyltransferases (2.7.7)
    • C12N9/1276RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07Nucleotidyltransferases (2.7.7)
    • C12Y207/07049RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
EP22702103.7A 2021-01-11 2022-01-11 Prime-editor-varianten, konstrukte und verfahren zur verbesserung der prime-editierungseffizienz und -präzision Pending EP4274894A2 (de)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US202163136194P 2021-01-11 2021-01-11
US202163176180P 2021-04-16 2021-04-16
US202163176202P 2021-04-16 2021-04-16
US202163194865P 2021-05-28 2021-05-28
US202163194913P 2021-05-28 2021-05-28
US202163231230P 2021-08-09 2021-08-09
US202163255897P 2021-10-14 2021-10-14
PCT/US2022/012054 WO2022150790A2 (en) 2021-01-11 2022-01-11 Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision

Publications (1)

Publication Number Publication Date
EP4274894A2 true EP4274894A2 (de) 2023-11-15

Family

ID=80119064

Family Applications (1)

Application Number Title Priority Date Filing Date
EP22702103.7A Pending EP4274894A2 (de) 2021-01-11 2022-01-11 Prime-editor-varianten, konstrukte und verfahren zur verbesserung der prime-editierungseffizienz und -präzision

Country Status (5)

Country Link
EP (1) EP4274894A2 (de)
JP (1) JP2024503437A (de)
AU (1) AU2022206476A1 (de)
CA (1) CA3203876A1 (de)
WO (1) WO2022150790A2 (de)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023096847A2 (en) * 2021-11-24 2023-06-01 Prime Medicine, Inc. Methods and compositions for inhibiting mismatch repair
US20240052370A1 (en) * 2022-07-27 2024-02-15 Inscripta, Inc. Modulating cellular repair mechanisms for genomic editing
WO2024077267A1 (en) 2022-10-07 2024-04-11 The Broad Institute, Inc. Prime editing methods and compositions for treating triplet repeat disorders

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4663290A (en) 1982-01-21 1987-05-05 Molecular Genetics, Inc. Production of reverse transcriptase
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5374553A (en) 1986-08-22 1994-12-20 Hoffmann-La Roche Inc. DNA encoding a thermostable nucleic acid polymerase enzyme from thermotoga maritima
US4889818A (en) 1986-08-22 1989-12-26 Cetus Corporation Purified thermostable enzyme
US5079352A (en) 1986-08-22 1992-01-07 Cetus Corporation Purified thermostable enzyme
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5244797B1 (en) 1988-01-13 1998-08-25 Life Technologies Inc Cloned genes encoding reverse transcriptase lacking rnase h activity
US4965185A (en) 1988-06-22 1990-10-23 Grischenko Valentin I Method for low-temperature preservation of embryos
US5270179A (en) 1989-08-10 1993-12-14 Life Technologies, Inc. Cloning and expression of T5 DNA polymerase reduced in 3'- to-5' exonuclease activity
US5047342A (en) 1989-08-10 1991-09-10 Life Technologies, Inc. Cloning and expression of T5 DNA polymerase
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
JP2709311B2 (ja) 1990-09-28 1998-02-04 エフ.ホフマン−ラ ロシュ アクチェンゲゼルシャフト 熱安定性dnaポリメラーゼの5→3′のエキソヌクレアーゼ突然変異
EP0553264A4 (en) 1990-10-05 1994-07-13 Wayne M Barnes Thermostable dna polymerase
US5436149A (en) 1993-02-19 1995-07-25 Barnes; Wayne M. Thermostable DNA polymerase with enhanced thermostability and enhanced length and efficiency of primer extension
US5512462A (en) 1994-02-25 1996-04-30 Hoffmann-La Roche Inc. Methods and reagents for the polymerase chain reaction amplification of long DNA sequences
US5912155A (en) 1994-09-30 1999-06-15 Life Technologies, Inc. Cloned DNA polymerases from Thermotoga neapolitana
US5614365A (en) 1994-10-17 1997-03-25 President & Fellow Of Harvard College DNA polymerase having modified nucleotide binding site for DNA sequencing
US5773258A (en) 1995-08-25 1998-06-30 Roche Molecular Systems, Inc. Nucleic acid amplification using a reversibly inactivated thermostable enzyme
US6183998B1 (en) 1998-05-29 2001-02-06 Qiagen Gmbh Max-Volmer-Strasse 4 Method for reversible modification of thermostable enzymes
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
GB9920194D0 (en) 1999-08-27 1999-10-27 Advanced Biotech Ltd A heat-stable thermostable DNA polymerase for use in nucleic acid amplification
AU785007B2 (en) 1999-11-24 2006-08-24 Mcs Micro Carrier Systems Gmbh Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells
US8178291B2 (en) 2005-02-18 2012-05-15 Monogram Biosciences, Inc. Methods and compositions for determining hypersusceptibility of HIV-1 to non-nucleoside reverse transcriptase inhibitors
US9783791B2 (en) 2005-08-10 2017-10-10 Agilent Technologies, Inc. Mutant reverse transcriptase and methods of use
EP2137300B1 (de) 2007-04-26 2011-10-26 Ramot at Tel-Aviv University Ltd. Pluripotente autologe Stammzellen aus der mündlichen oder gastrointestinalen Schleimhaut
CA3073384A1 (en) 2008-09-05 2010-03-11 President And Fellows Of Harvard College Continuous directed evolution of proteins and nucleic acids
EP3415621A1 (de) 2009-03-04 2018-12-19 Board Of Regents The University Of Texas System Stabilisierte reverse-transkriptase-fusionsproteine
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
SG181601A1 (en) 2009-12-10 2012-07-30 Univ Minnesota Tal effector-mediated dna modification
WO2012054727A1 (en) 2010-10-22 2012-04-26 Bio-Rad Laboratories, Inc. Reverse transcriptase mixtures with improved storage stability
EP2655614B1 (de) 2010-12-22 2017-03-15 President and Fellows of Harvard College Kontinuierliche gerichtete evolution
AU2012314355B2 (en) 2011-09-28 2018-01-18 Zera Intein Protein Solutions, S.L. Split inteins and uses thereof
PL2877490T3 (pl) 2012-06-27 2019-03-29 The Trustees Of Princeton University Inteiny podzielone, koniugaty i ich zastosowania
US9181535B2 (en) 2012-09-24 2015-11-10 The Chinese University Of Hong Kong Transcription activator-like effector nucleases (TALENs)
WO2014055778A2 (en) 2012-10-03 2014-04-10 Agrivida, Inc. Multiprotein expression cassettes
JO3470B1 (ar) 2012-10-08 2020-07-05 Merck Sharp & Dohme مشتقات 5- فينوكسي-3h-بيريميدين-4-أون واستخدامها كمثبطات ناسخ عكسي ل hiv
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2015134121A2 (en) 2014-01-20 2015-09-11 President And Fellows Of Harvard College Negative selection and stringency modulation in continuous evolution systems
DK3212778T3 (da) 2014-10-28 2019-11-04 Agrivida Inc Fremgangsmåder og sammensætninger til stabilisering af trans-splejsning af intein-modificerede proteaser
WO2016168631A1 (en) 2015-04-17 2016-10-20 President And Fellows Of Harvard College Vector-based mutagenesis system
US9580698B1 (en) 2016-09-23 2017-02-28 New England Biolabs, Inc. Mutant reverse transcriptase
JP2022518256A (ja) * 2019-01-29 2022-03-14 ユニバーシティ オブ ワシントン 遺伝子編集の方法
US20220177877A1 (en) * 2019-03-04 2022-06-09 President And Fellows Of Harvard College Highly multiplexed base editing
JP2022526908A (ja) * 2019-03-19 2022-05-27 ザ ブロード インスティテュート,インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物

Also Published As

Publication number Publication date
AU2022206476A1 (en) 2023-07-20
WO2022150790A3 (en) 2022-08-11
WO2022150790A2 (en) 2022-07-14
JP2024503437A (ja) 2024-01-25
CA3203876A1 (en) 2022-07-14

Similar Documents

Publication Publication Date Title
US11643652B2 (en) Methods and compositions for prime editing nucleotide sequences
JP2023525304A (ja) 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
US20230021641A1 (en) Cas9 variants having non-canonical pam specificities and uses thereof
JP2023543803A (ja) プライム編集ガイドrna、その組成物、及びその使用方法
JPWO2020191233A5 (de)
JPWO2020191234A5 (de)
JPWO2020191243A5 (de)
WO2022150790A2 (en) Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision
WO2023076898A1 (en) Methods and compositions for editing a genome with prime editing and a recombinase
WO2023015309A2 (en) Improved prime editors and methods of use
CN117321201A (zh) 用于增强引导编辑效率和精度的引导编辑器变体、构建体和方法
WO2023205687A1 (en) Improved prime editing methods and compositions
CN116685682A (zh) 引导编辑向导rna、其组合物以及使用它们的方法

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: UNKNOWN

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20230717

AK Designated contracting states

Kind code of ref document: A2

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)