BR112020024863A2 - Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso - Google Patents

Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso

Info

Publication number
BR112020024863A2
BR112020024863A2 BR112020024863A BR112020024863A BR112020024863A2 BR 112020024863 A2 BR112020024863 A2 BR 112020024863A2 BR 112020024863 A BR112020024863 A BR 112020024863A BR 112020024863 A BR112020024863 A BR 112020024863A BR 112020024863 A2 BR112020024863 A2 BR 112020024863A2
Authority
BR
Brazil
Prior art keywords
rna
interest
methods
target sequence
crispr
Prior art date
Application number
BR112020024863A
Other languages
English (en)
Inventor
Briner Crawley Alexandra
Michael Coyle
Rodolphe Barrangou
D Elich Tedd
D Bowen Tyson
Original Assignee
Lifeedit 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 Lifeedit Inc filed Critical Lifeedit Inc
Publication of BR112020024863A2 publication Critical patent/BR112020024863A2/pt

Links

Classifications

    • 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
    • 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
    • C12N15/10—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
    • 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
    • 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
    • C12N15/113—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
    • 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
    • C12N15/63—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 RNAses, DNAses
    • 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 [CRISPRs]
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso. são fornecidas composições e métodos de ligação a uma sequência alvo de interesse. as composições são úteis para clivar ou modificar uma sequência alvo de interesse, visualizar uma sequência alvo de interesse e modificar a expressão de uma sequência de interesse. as composições compreendem polipeptídeos de nuclease guiada por rna, rnas de crispr, rnas transativadores de crispr, rnas-guias, bem como moléculas de ácido nucleico que codificam os mesmos. também são fornecidos os vetores e as células hospedeiras que compreendem as moléculas de ácido nucleico. são fornecidos, ainda, sistemas de crispr para ligar uma sequência alvo de interesse, em que o sistema de crispr compreende um polipeptídeo de nuclease guiada por rna e um ou mais rnas-guias.
BR112020024863A 2018-06-05 2019-06-04 Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso BR112020024863A2 (pt)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US201862680859P 2018-06-05 2018-06-05
US201862680846P 2018-06-05 2018-06-05
US201862680853P 2018-06-05 2018-06-05
US201862680863P 2018-06-05 2018-06-05
US201862680862P 2018-06-05 2018-06-05
US201862680845P 2018-06-05 2018-06-05
US201862686901P 2018-06-19 2018-06-19
US201962805045P 2019-02-13 2019-02-13
US201962805041P 2019-02-13 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

Publications (1)

Publication Number Publication Date
BR112020024863A2 true BR112020024863A2 (pt) 2022-02-01

Family

ID=67002401

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112020024863A BR112020024863A2 (pt) 2018-06-05 2019-06-04 Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso

Country Status (14)

Country Link
US (2) US11162114B2 (pt)
EP (1) EP3802807A1 (pt)
JP (1) JP2021526853A (pt)
KR (1) KR20210089629A (pt)
CN (1) CN113015797A (pt)
AU (1) AU2019282149A1 (pt)
BR (1) BR112020024863A2 (pt)
CA (1) CA3102840A1 (pt)
IL (1) IL279222A (pt)
MX (1) MX2020013124A (pt)
SG (1) SG11202011975WA (pt)
TW (1) TW202010843A (pt)
WO (1) WO2019236566A1 (pt)
ZA (1) ZA202008045B (pt)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3033327A1 (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
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
KR20190130613A (ko) 2017-03-23 2019-11-22 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC 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
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
EP3942040A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
MX2022001849A (es) 2019-08-12 2022-03-11 Lifeedit Therapeutics Inc Nucleasas guiadas por acido ribonucleico (arn) y sus fragmentos activos y variantes y metodos de uso.
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
WO2021222318A1 (en) * 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
GB202007943D0 (en) 2020-05-27 2020-07-08 Snipr Biome Aps Products & methods
IL299261A (en) 2020-07-01 2023-02-01 Elevatebio Tech Inc Compositions and methods for reprogramming cells using circular RNA
CA3173949A1 (en) 2020-07-15 2022-01-20 LifeEDIT Therapeutics, Inc. Uracil stabilizing proteins and active fragments and variants thereof and methods of use
WO2022027035A1 (en) * 2020-07-27 2022-02-03 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
JP2023541400A (ja) 2020-09-11 2023-10-02 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 Ｄｎａ修飾酵素並びにその活性断片及びバリアント並びにその使用方法
KR20240011120A (ko) 2020-12-22 2024-01-25 크로마 메디슨, 인크. 후성유전학적 편집을 위한 조성물 및 방법
TW202300649A (zh) 2021-03-22 2023-01-01 美商生命編輯治療學公司 Ｄｎａ修飾酶及活性片段及其變體及使用方法
CA3173953A1 (en) 2021-06-11 2023-12-10 Tyson D. BOWEN Rna polymerase iii promoters and methods of use
WO2023164591A2 (en) * 2022-02-23 2023-08-31 Metagenomi, Inc. Systems and methods for transposing cargo nucleotide sequences
WO2023215711A1 (en) 2022-05-01 2023-11-09 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of pcsk9 expression
WO2023250509A1 (en) 2022-06-23 2023-12-28 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of b2m expression
WO2023250512A1 (en) 2022-06-23 2023-12-28 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of ciita expression
WO2023250490A1 (en) 2022-06-23 2023-12-28 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of trac expression
WO2024042489A1 (en) 2022-08-25 2024-02-29 LifeEDIT Therapeutics, Inc. Chemical modification of guide rnas with 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
WO2024064910A1 (en) 2022-09-23 2024-03-28 Chroma Medicine, 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
WO2024095245A2 (en) 2022-11-04 2024-05-10 LifeEDIT Therapeutics, Inc. Evolved adenine deaminases and rna-guided nuclease fusion proteins with internal insertion sites and methods of use

Family Cites Families (65)

* 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
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
JP3046063B2 (ja) 1989-03-17 2000-05-29 イー・アイ・デユポン・ドウ・ヌムール・アンド・カンパニー 遺伝子の発現の外部の調節
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
WO1993004177A1 (en) 1991-08-27 1993-03-04 Agricultural Genetics Company Limited Proteins with insecticidal properties against homopteran insects and their use in plant protection
TW261517B (pt) 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
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
US5789156A (en) 1993-06-14 1998-08-04 Basf Ag Tetracycline-regulated transcriptional inhibitors
US5837458A (en) 1994-02-17 1998-11-17 Maxygen, Inc. Methods and compositions for cellular and metabolic engineering
US5605793A (en) 1994-02-17 1997-02-25 Affymax Technologies N.V. Methods for in vitro recombination
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
AU762993C (en) 1998-02-26 2004-06-10 Pioneer Hi-Bred International, Inc. Constitutive maize promoters
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
CN1360632A (zh) 1999-05-04 2002-07-24 孟山都技术有限公司 鞘翅目毒性多肽组合物和抗虫转基因植物
CA2384967A1 (en) 1999-09-15 2001-03-22 Monsanto Technology Llc Lepidopteran-active bacillus thuringiensis .delta.-endotoxin compositions and methods of use
EP2359869B1 (en) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
US20050183161A1 (en) 2003-10-14 2005-08-18 Athenix Corporation AXMI-010, a delta-endotoxin gene and methods for its use
US7629504B2 (en) 2003-12-22 2009-12-08 Pioneer Hi-Bred International, Inc. Bacillus thuringiensis cry9 nucleic acids
ES2397118T3 (es) 2006-06-14 2013-03-04 Athenix Corporation AXMI-031, AXMI-039 ,AXMI-040 y AXMI-049, una familia de genes de endotoxina delta y métodos para su uso
PT2334794T (pt) 2008-09-15 2017-02-15 Harvard College Modulação de bcl11a para tratamento de hemoglobinopatias
WO2011002992A1 (en) 2009-07-02 2011-01-06 Athenix Corp. Axmi-205 pesticidal gene and methods for its use
WO2011084324A2 (en) 2009-12-21 2011-07-14 Pioneer Hi-Bred International, Inc. Novel bacillus thuringiensis gene with lepidopteran activity
CN103154247A (zh) 2010-08-19 2013-06-12 先锋国际良种公司 对鳞翅目昆虫具有活性的新苏云金杆菌基因
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
PT2800811T (pt) * 2012-05-25 2017-08-17 Univ California Métodos e composições para modificação de adn alvo dirigida por arn e para modulação dirigida por arn de transcrição
HUE030799T2 (en) 2012-07-11 2017-06-28 Sangamo Biosciences Inc Methods and preparations for the treatment of lysosomal storage diseases
CA2930877A1 (en) * 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
WO2016033298A1 (en) 2014-08-28 2016-03-03 North Carolina State University Novel cas9 proteins and guiding features 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
IL294014B1 (en) 2015-10-23 2024-03-01 Harvard College Nucleobase editors and their uses
EP3426778A1 (en) * 2016-03-11 2019-01-16 Pioneer Hi-Bred International, Inc. Novel cas9 systems and methods of use
EP3699281A1 (en) * 2016-03-11 2020-08-26 Pioneer Hi-Bred International, Inc. Novel cas9 systems and methods of use
EP3472310A1 (en) * 2016-06-20 2019-04-24 Pioneer Hi-Bred International, Inc. Novel cas systems and methods of use
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
WO2018035250A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
CN110214180A (zh) * 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送

Also Published As

Publication number Publication date
JP2021526853A (ja) 2021-10-11
TW202010843A (zh) 2020-03-16
ZA202008045B (en) 2023-04-26
US20190367949A1 (en) 2019-12-05
WO2019236566A1 (en) 2019-12-12
CN113015797A (zh) 2021-06-22
KR20210089629A (ko) 2021-07-16
MX2020013124A (es) 2021-07-15
US11926843B2 (en) 2024-03-12
AU2019282149A1 (en) 2021-01-21
US11162114B2 (en) 2021-11-02
IL279222A (en) 2021-01-31
US20220002756A1 (en) 2022-01-06
EP3802807A1 (en) 2021-04-14
CA3102840A1 (en) 2019-12-12
SG11202011975WA (en) 2020-12-30

Similar Documents

Publication Publication Date Title
BR112020024863A2 (pt) Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso
BR112022002695A2 (pt) Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso
BR112021012665A2 (pt) Polipeptídeos úteis para edição de genes e métodos de uso
BR112022021459A2 (pt) Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso
BR112019006388A2 (pt) enzimas modificadoras de ácido nucleico guiadas por rna e métodos de uso das mesmas
BR112019006384A2 (pt) enzimas modificadoras de ácido nucleico guiadas por rna e métodos de uso das mesmas
MX2020004578A (es) Composiciones de casz y metodos de uso.
BR112021020608A8 (pt) Rna recrutador de desaminase, construção, método para editar um rna alvo em uma célula hospedeira, e, construção, composição, célula, biblioteca ou kit
BR112019007433A2 (pt) sistemas de replicon de vírus recombinante e usos dos mesmos
BR112018074463A2 (pt) anticorpos anti-tim-3 e métodos de uso dos mesmos.
BR112018068189A2 (pt) proteínas de ligação induzíveis e métodos de uso
SA517390590B1 (ar) Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة
AU2016262093A8 (en) Enhancing endonuclease based gene editing in primary cells
BR112015016005A2 (pt) preparação de amostra em um suporte sólido
ES2690386T3 (es) Composición para escindir un ADN diana que comprende un ARN guía específico para el ADN diana y el ácido nucleico que codifica la proteína Cas o la propia proteína Cas, y sus usos
CL2021000028A1 (es) Anticuerpo multiespecifico (divisional de solicitud 937-2020).
MX2020005282A (es) Vector para la produccion de particulas aav.
BRPI0517121A (pt) célula hospedeira eucariótica de planta não-superior que contém proteìna de fusão recombinante dentro de conjuntos do tipo corpo de proteìna recombinante ( rpblas ), método para a produção de uma proteìna de fusão e molécula de ácido nucléico recombinante
BR112018075855A2 (pt) sistema de vetor viral adeno-associado com dupla sobreposição para a expressão abc4a
BR112017018149A2 (pt) célula hospedeira recombinante, método para produzir uma proteína de interesse recombinante, sistema de expressão, e, processos para fabricar uma formulação de proteína estável e para reduzir a atividade de esterase em uma formulação de proteína.
BR112015022641A2 (pt) construção de dna, célula hospedeira eucariótica, linhagem de células eucarióticas e processo de preparação de uma proteína-alvo
BR112015019719A2 (pt) Proteínas melhoradas de ligação ao tnf
BR112023022367A2 (pt) Anticorpos anti-tigit e métodos de uso dos mesmos
EA202092929A1 (ru) Рнк-управляемые нуклеазы и активные фрагменты и их варианты и способы применения
BR112023023260A2 (pt) Vetor, métodos para produzir um vetor e para tratar um sujeito tendo uma doença ou uma doença infecciosa, célula hospedeira, e, composição farmacêutica