AU2019282149B2 - RNA-guided nucleases and active fragments and variants thereof and methods of use - Google Patents

RNA-guided nucleases and active fragments and variants thereof and methods of use

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Publication number
AU2019282149B2
AU2019282149B2 AU2019282149A AU2019282149A AU2019282149B2 AU 2019282149 B2 AU2019282149 B2 AU 2019282149B2 AU 2019282149 A AU2019282149 A AU 2019282149A AU 2019282149 A AU2019282149 A AU 2019282149A AU 2019282149 B2 AU2019282149 B2 AU 2019282149B2
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sequence
polypeptide
rgn
seq
rna
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AU2019282149A
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AU2019282149A1 (en
Inventor
Rodolphe Barrangou
Tyson D. BOWEN
Michael Coyle
Alexandra Briner CRAWLEY
Tedd D. Elich
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Life Edit Therapeutics Inc
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Life Edit Therapeutics Inc
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Assigned to LifeEDIT Therapeutics, Inc. reassignment LifeEDIT Therapeutics, Inc. Amend patent request/document other than specification (104) Assignors: LIFEEDIT, INC.
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Publication of AU2019282149B2 publication Critical patent/AU2019282149B2/en
Assigned to LIFE EDIT THERAPEUTICS, INC. reassignment LIFE EDIT THERAPEUTICS, INC. Amend patent request/document other than specification (104) Assignors: LifeEDIT Therapeutics, Inc.
Priority to AU2025271070A priority Critical patent/AU2025271070A1/en
Active legal-status Critical Current
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2521/00—Reaction characterised by the enzymatic activity
    • C12Q2521/30—Phosphoric diester hydrolysing, i.e. nuclease
    • C12Q2521/301—Endonuclease

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
AU2019282149A 2018-06-05 2019-06-04 RNA-guided nucleases and active fragments and variants thereof and methods of use Active AU2019282149B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025271070A AU2025271070A1 (en) 2018-06-05 2025-11-20 RNA-guided nucleases and active fragments and variants thereof and methods of use

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
US201862680862P 2018-06-05 2018-06-05
US201862680845P 2018-06-05 2018-06-05
US201862680846P 2018-06-05 2018-06-05
US201862680859P 2018-06-05 2018-06-05
US201862680853P 2018-06-05 2018-06-05
US201862680863P 2018-06-05 2018-06-05
US62/680,846 2018-06-05
US62/680,862 2018-06-05
US62/680,863 2018-06-05
US62/680,859 2018-06-05
US62/680,845 2018-06-05
US62/680,853 2018-06-05
US201862686901P 2018-06-19 2018-06-19
US62/686,901 2018-06-19
US201962805041P 2019-02-13 2019-02-13
US201962805045P 2019-02-13 2019-02-13
US62/805,041 2019-02-13
US62/805,045 2019-02-13
PCT/US2019/035373 WO2019236566A1 (en) 2018-06-05 2019-06-04 Rna-guided nucleases and active fragments and variants thereof and methods of use

Related Child Applications (1)

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AU2025271070A Division AU2025271070A1 (en) 2018-06-05 2025-11-20 RNA-guided nucleases and active fragments and variants thereof and methods of use

Publications (2)

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AU2019282149A1 AU2019282149A1 (en) 2021-01-21
AU2019282149B2 true AU2019282149B2 (en) 2025-08-21

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AU2025271070A Pending AU2025271070A1 (en) 2018-06-05 2025-11-20 RNA-guided nucleases and active fragments and variants thereof and methods of use

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US (4) US11162114B2 (enExample)
EP (2) EP3802807B1 (enExample)
JP (2) JP7708660B2 (enExample)
KR (1) KR20210089629A (enExample)
CN (1) CN113015797A (enExample)
AU (2) AU2019282149B2 (enExample)
BR (1) BR112020024863A2 (enExample)
CA (1) CA3102840A1 (enExample)
DK (1) DK3802807T3 (enExample)
ES (1) ES3011849T3 (enExample)
FI (1) FI3802807T3 (enExample)
IL (2) IL279222B2 (enExample)
MX (1) MX2020013124A (enExample)
PL (1) PL3802807T3 (enExample)
PT (1) PT3802807T (enExample)
SG (1) SG11202011975WA (enExample)
TW (1) TW202532650A (enExample)
WO (1) WO2019236566A1 (enExample)
ZA (1) ZA202008045B (enExample)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2021030344A1 (en) 2019-08-12 2021-02-18 Lifeedit, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3157131A1 (en) * 2019-12-10 2021-06-17 Inscripta, Inc. Novel mad nucleases
JP2023508731A (ja) * 2019-12-30 2023-03-03 ライフエディット セラピューティクス，インコーポレイティド Ｒｎａ誘導ヌクレアーゼ、その活性断片および多様体、ならびに使用方法
TW202208626A (zh) 2020-04-24 2022-03-01 美商生命編輯公司 Rna引導核酸酶及其活性片段與變體，以及使用方法
EP4143315A1 (en) * 2020-04-28 2023-03-08 The Broad Institute Inc. <smallcaps/>? ? ?ush2a? ? ? ? ?targeted base editing of thegene
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
GB202007943D0 (en) 2020-05-27 2020-07-08 Snipr Biome Aps Products & methods
AU2021300378A1 (en) 2020-07-01 2023-01-19 Elevatebio Technologies, Inc. Compositions and methods for cellular reprogramming using circular RNA
US20240247242A1 (en) 2020-07-15 2024-07-25 LifeEDIT Therapeutics, Inc. Uracil stabilizing proteins and active fragments and variants thereof and methods of use
US20230340486A1 (en) * 2020-07-27 2023-10-26 The Children’S Hospital Of Philadelphia In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders
MX2023002848A (es) 2020-09-11 2023-03-22 Lifeedit Therapeutics Inc Enzimas modificadoras del acido desoxirribonucleico (adn) y fragmentos activos y sus variantes y metodos de uso.
KR20240011120A (ko) 2020-12-22 2024-01-25 크로마 메디슨, 인크. 후성유전학적 편집을 위한 조성물 및 방법
US20240301385A1 (en) 2021-03-22 2024-09-12 LifeEDIT Therapeutics, Inc. Dna modifying enzymes and active fragments and variants thereof and methods of use
EP4352215A1 (en) 2021-06-11 2024-04-17 LifeEDIT Therapeutics, Inc. Rna polymerase iii promoters and methods of use
AU2023225035A1 (en) * 2022-02-23 2024-09-05 Metagenomi Therapeutics, Inc. Systems and methods for transposing cargo nucleotide sequences
CN114634923B (zh) * 2022-04-07 2024-02-23 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
IL316722A (en) 2022-05-01 2024-12-01 Chroma Medicine Inc Compositions and methods for epigenetic regulation of PCSK9 expression
JP2025524455A (ja) 2022-06-23 2025-07-30 エヌクロマ・バイオ，インコーポレーテッド Ｔｒａｃ発現のエピジェネティック調節のための組成物及び方法
EP4544058A1 (en) 2022-06-23 2025-04-30 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of ciita expression
WO2023250148A1 (en) 2022-06-23 2023-12-28 Chroma Medicine, Inc. Compositions and methods for epigenetic editing
US20250387518A1 (en) 2022-06-23 2025-12-25 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of b2m expression
CA3265664A1 (en) 2022-08-25 2024-02-29 Life Edit Therapeutics, Inc. CHEMICAL MODIFICATION OF GUIDE RNA WITH A LOCKED NUCLEIC ACID FOR RNA-GUIDED NUCLEASE-MEDIATED GENE EDITING
CN115820636B (zh) * 2022-09-02 2024-06-11 华南农业大学 靶向TP53基因的sgRNA及其应用
IL319704A (en) 2022-09-23 2025-05-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of HBV gene expression
WO2024081879A1 (en) 2022-10-14 2024-04-18 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of cd247 expression
KR20250126155A (ko) 2022-11-04 2025-08-22 라이프 에디트 테라퓨틱스, 인크. 내부 삽입 부위를 갖는 진화된 아데닌 데아미나제 및 rna-가이드 뉴클레아제 융합 단백질 및 사용 방법
WO2024178397A2 (en) 2023-02-24 2024-08-29 Elevatebio Technologies, Inc. Modified immune effector cells and methods of use
WO2024238700A1 (en) 2023-05-15 2024-11-21 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2025174908A1 (en) 2024-02-12 2025-08-21 Life Edit Therapeutics, Inc. Novel rna-guided nucleases and proteins for polymerase editing
WO2026003754A1 (en) 2024-06-25 2026-01-02 Life Edit Therapeutics, Inc. Novel reverse transcriptases and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013176772A1 (en) * 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2017155714A1 (en) * 2016-03-11 2017-09-14 Pioneer Hi-Bred International, Inc. Novel cas9 systems and methods of use

Family Cites Families (69)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US457A (en) 1837-11-11 Caleb slade
US10167A (en) 1853-10-25 Soda-jountaiet
US699A (en) 1838-04-21 stone
US9840A (en) 1853-07-12 Improvement in tanning
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4196265A (en) 1977-06-15 1980-04-01 The Wistar Institute Method of producing antibodies
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
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
US5380831A (en) 1986-04-04 1995-01-10 Mycogen Plant Science, Inc. Synthetic insecticidal crystal protein gene
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
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5569597A (en) 1985-05-13 1996-10-29 Ciba Geigy Corp. Methods of inserting viral DNA into plant material
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US4853331A (en) 1985-08-16 1989-08-01 Mycogen Corporation Cloning and expression of Bacillus thuringiensis toxin gene toxic to beetles of the order Coleoptera
US5268463A (en) 1986-11-11 1993-12-07 Jefferson Richard A Plant promoter α-glucuronidase gene construct
US5608142A (en) 1986-12-03 1997-03-04 Agracetus, Inc. Insecticidal cotton plants
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5039523A (en) 1988-10-27 1991-08-13 Mycogen Corporation Novel Bacillus thuringiensis isolate denoted B.t. PS81F, active against lepidopteran pests, and a gene encoding a lepidopteran-active toxin
EP0388186A1 (en) 1989-03-17 1990-09-19 E.I. Du Pont De Nemours And Company External regulation of gene expression
ATE225853T1 (de) 1990-04-12 2002-10-15 Syngenta Participations Ag Gewebe-spezifische promotoren
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
US5498830A (en) 1990-06-18 1996-03-12 Monsanto Company Decreased oil content in plant seeds
CA2051562C (en) 1990-10-12 2003-12-02 Jewel M. Payne Bacillus thuringiensis isolates active against dipteran pests
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5399680A (en) 1991-05-22 1995-03-21 The Salk Institute For Biological Studies Rice chitinase promoter
US5604121A (en) 1991-08-27 1997-02-18 Agricultural Genetics Company Limited Proteins with insecticidal properties against homopteran insects and their use in plant protection
TW261517B (enExample) 1991-11-29 1995-11-01 Mitsubishi Shozi Kk
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5789156A (en) 1993-06-14 1998-08-04 Basf Ag Tetracycline-regulated transcriptional inhibitors
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
US5605793A (en) 1994-02-17 1997-02-25 Affymax Technologies N.V. Methods for in vitro recombination
US5837458A (en) 1994-02-17 1998-11-17 Maxygen, Inc. Methods and compositions for cellular and metabolic engineering
US5608144A (en) 1994-08-12 1997-03-04 Dna Plant Technology Corp. Plant group 2 promoters and uses thereof
US5659026A (en) 1995-03-24 1997-08-19 Pioneer Hi-Bred International ALS3 promoter
US6072050A (en) 1996-06-11 2000-06-06 Pioneer Hi-Bred International, Inc. Synthetic promoters
AR014072A1 (es) 1998-02-26 2001-01-31 Pioneer Hi Bred Int Molecula de acido nucleico aislada que tiene una secuencia nucleotidica para un promotor que es capaz de iniciar una transcripcion constitutiva en unacelula de planta, construccion adn, vector, celula huesped, metodo para expresar en forma constitutiva una secuencia nucleotidica heteroloca en una
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6555655B1 (en) 1999-05-04 2003-04-29 Monsanto Technology, Llc Coleopteran-toxic polypeptide compositions and insect-resistant transgenic plants
US6593293B1 (en) 1999-09-15 2003-07-15 Monsanto Technology, Llc Lepidopteran-active Bacillus thuringiensis δ-endotoxin compositions and methods of use
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
US20050183161A1 (en) 2003-10-14 2005-08-18 Athenix Corporation AXMI-010, a delta-endotoxin gene and methods for its use
WO2005066202A2 (en) 2003-12-22 2005-07-21 E.I. Du Pont De Nemours And Company Bacillus cry9 family members
AU2007260716B2 (en) 2006-06-14 2013-05-02 BASF Agricultural Solutions Seed US LLC Axmi-031, axmi-039, axmi-040 and axmi-049, a family of delta-endotoxin genes and methods for their use
US8383604B2 (en) 2008-09-15 2013-02-26 Children's Medical Center Corporation Modulation of BCL11A for treatment of hemoglobinopathies
ES2609332T3 (es) 2009-07-02 2017-04-19 Athenix Corporation Gen pesticida AXMI-205 y métodos para su uso
WO2011084324A2 (en) 2009-12-21 2011-07-14 Pioneer Hi-Bred International, Inc. Novel bacillus thuringiensis gene with lepidopteran activity
WO2012024200A2 (en) 2010-08-19 2012-02-23 Pioneer Hi-Bred International, Inc. Novel bacillus thuringiensis gene with lepidopteran activity
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
EP3196301B1 (en) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of monogenic diseases
CA2930877A1 (en) * 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3186375A4 (en) 2014-08-28 2019-03-13 North Carolina State University Novel CAS9 PROTEINS AND CHARACTERISTICS FOR DNA TARGETING AND GENOME EDITING
AU2016263026A1 (en) * 2015-05-15 2017-11-09 Pioneer Hi-Bred International, Inc. Guide RNA/Cas endonuclease systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CN116555254A (zh) * 2015-10-09 2023-08-08 孟山都技术公司 新颖的rna导向性核酸酶及其用途
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
EP3426778A1 (en) * 2016-03-11 2019-01-16 Pioneer Hi-Bred International, Inc. Novel cas9 systems and methods of use
WO2017222773A1 (en) * 2016-06-20 2017-12-28 Pioneer Hi-Bred International, Inc. Novel cas systems and methods of use
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
EP3500967A1 (en) * 2016-08-17 2019-06-26 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
KR102622411B1 (ko) * 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
EP3538561A4 (en) * 2016-11-11 2020-10-21 The Regents of The University of California RNA-GUIDED VARIANT POLYPEPTIDES AND THEIR METHODS OF USE
KR20210149686A (ko) * 2018-12-27 2021-12-09 라이프에디트 테라퓨틱스, 인크. 유전자 편집에 유용한 폴리펩티드 및 사용 방법

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013176772A1 (en) * 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2017155714A1 (en) * 2016-03-11 2017-09-14 Pioneer Hi-Bred International, Inc. Novel cas9 systems and methods of use

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