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MX2024005318A - Plataforma de construccion unica para el suministro simultaneo de maquinaria de edicion de genes y carga de acido nucleico. - Google Patents

Plataforma de construccion unica para el suministro simultaneo de maquinaria de edicion de genes y carga de acido nucleico.

Info

Publication number
MX2024005318A
MX2024005318A MX2024005318A MX2024005318A MX2024005318A MX 2024005318 A MX2024005318 A MX 2024005318A MX 2024005318 A MX2024005318 A MX 2024005318A MX 2024005318 A MX2024005318 A MX 2024005318A MX 2024005318 A MX2024005318 A MX 2024005318A
Authority
MX
Mexico
Prior art keywords
nucleic acid
gene editing
simultaneous delivery
single construct
acid cargo
Prior art date
Application number
MX2024005318A
Other languages
English (en)
Inventor
Rahul Kakkar
Jonathan Douglas Finn
Brett Joseph Gordon Estes
Yijun Zhang
Original Assignee
Tome Biosciences 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 Tome Biosciences Inc filed Critical Tome Biosciences Inc
Publication of MX2024005318A publication Critical patent/MX2024005318A/es

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    • CCHEMISTRY; METALLURGY
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    • 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
    • C12N15/113Non-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
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    • 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
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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
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    • 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)
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    • 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
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    • 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]
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    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/30Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)

Abstract

En la presente descripción se proporcionan composiciones de ácidos nucleicos, métodos y una plataforma general para la ingeniería genética con especificidad de sitio mediante el uso de la adición programable mediante elementos de direccionamiento con especificidad de sitio (PASTE, por su sigla en inglés), edición de genes mediada por transposones u otra tecnología adecuada de edición o incorporación de genes empaquetada en una única construcción de ácido nucleico.
MX2024005318A 2021-11-01 2022-11-01 Plataforma de construccion unica para el suministro simultaneo de maquinaria de edicion de genes y carga de acido nucleico. MX2024005318A (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202163274483P 2021-11-01 2021-11-01
US202163282055P 2021-11-22 2021-11-22
US202263298941P 2022-01-12 2022-01-12
US202263318344P 2022-03-09 2022-03-09
US202263352897P 2022-06-16 2022-06-16
PCT/US2022/079035 WO2023077148A1 (en) 2021-11-01 2022-11-01 Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo

Publications (1)

Publication Number Publication Date
MX2024005318A true MX2024005318A (es) 2024-09-23

Family

ID=84767092

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024005318A MX2024005318A (es) 2021-11-01 2022-11-01 Plataforma de construccion unica para el suministro simultaneo de maquinaria de edicion de genes y carga de acido nucleico.

Country Status (7)

Country Link
EP (1) EP4426828A1 (es)
KR (1) KR20240099393A (es)
AU (1) AU2022375820A1 (es)
CA (1) CA3237300A1 (es)
IL (1) IL312452A (es)
MX (1) MX2024005318A (es)
WO (1) WO2023077148A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (107)

* 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
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
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
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
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
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
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
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
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
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
US6027726A (en) 1994-09-30 2000-02-22 Inex Phamaceuticals Corp. Glycosylated protein-liposome conjugates and methods for their preparation
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
NZ504214A (en) 1997-10-24 2003-06-30 Invitrogen Corp Recombination cloning using nucleic acids having recombination sites
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
JP4722481B2 (ja) 2002-06-28 2011-07-13 プロティバ バイオセラピューティクス リミテッド リポソーム製造方法および装置
BRPI0513390A (pt) 2004-07-16 2008-05-06 Us Gov Health & Human Serv vacinas contra aids contendo construções de ácido nucléico cmv/r
AU2006274413B2 (en) 2005-07-27 2013-01-10 Arbutus Biopharma Corporation Systems and methods for manufacturing liposomes
CA2711179A1 (en) 2007-12-31 2009-07-16 Nanocor Therapeutics, Inc. Rna interference for the treatment of heart failure
NZ716192A (en) 2009-12-01 2017-07-28 Shire Human Genetic Therapies Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
US8691750B2 (en) 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
KR102128248B1 (ko) 2011-06-08 2020-07-01 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
AU2012333134B2 (en) 2011-07-22 2017-05-25 John Paul Guilinger Evaluation and improvement of nuclease cleavage specificity
WO2013039861A2 (en) 2011-09-12 2013-03-21 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof
WO2013039857A1 (en) 2011-09-12 2013-03-21 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof
DK3988537T1 (da) 2011-12-07 2022-05-23 Alnylam Pharmaceuticals Inc Bionedbrydelige lipider til afgivelse af aktive midler
US11021737B2 (en) 2011-12-22 2021-06-01 President And Fellows Of Harvard College Compositions and methods for analyte detection
WO2013116126A1 (en) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Novel low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
ES2636902T3 (es) 2012-05-25 2017-10-10 The Regents Of The University Of California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
SG10201809566SA (en) 2012-10-23 2018-11-29 Toolgen Inc Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
CA2891347C (en) 2012-12-06 2018-02-27 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
KR20150105633A (ko) 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 시스템, 방법 및 최적화된 가이드 조성물의 조작
WO2014093595A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
CN113355357A (zh) 2012-12-12 2021-09-07 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
EP3327127B1 (en) 2012-12-12 2021-03-24 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
SG11201504621RA (en) 2012-12-17 2015-07-30 Harvard College Rna-guided human genome engineering
EP2970923B1 (en) 2013-03-13 2018-04-11 President and Fellows of Harvard College Mutants of cre recombinase
EP2970940B1 (en) 2013-03-14 2018-07-25 Translate Bio, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
JP6738728B2 (ja) 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのCRISPR−Cas系、ベクターおよび組成物の送達および使用
SG11201510286QA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
KR20160044457A (ko) 2013-06-17 2016-04-25 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 탠덤 안내 시스템, 방법 및 조성물의 전달, 조작 및 최적화
JP6718813B2 (ja) 2013-07-10 2020-07-08 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 Rna誘導性遺伝子制御および編集のための直交性cas9タンパク質
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
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
US9388430B2 (en) * 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2015056756A1 (ja) 2013-10-18 2015-04-23 国立大学法人熊本大学 多能性幹細胞からの腎臓誘導法
CA2930015A1 (en) 2013-11-07 2015-05-14 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
CN106029880A (zh) 2013-12-12 2016-10-12 布罗德研究所有限公司 核苷酸重复障碍中CRISPR-Cas系统的组合物和使用方法
WO2015089427A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物
RS58337B1 (sr) 2014-03-24 2019-03-29 Translate Bio Inc Irnk terapija za lečenje očnih oboljenja
AU2015283954B2 (en) 2014-07-02 2020-11-12 Translate Bio, Inc. Encapsulation of messenger RNA
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CN113789317B (zh) 2014-08-06 2024-02-23 基因工具股份有限公司 使用空肠弯曲杆菌crispr/cas系统衍生的rna引导的工程化核酸酶的基因编辑
EP3189140B1 (en) 2014-09-05 2019-10-23 Vilnius University Programmable rna shredding by the type iii-a crispr-cas system of streptococcus thermophilus
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
US11071790B2 (en) 2014-10-29 2021-07-27 Massachusetts Eye And Ear Infirmary Method for efficient delivery of therapeutic molecules in vitro and in vivo
WO2016070136A1 (en) 2014-10-31 2016-05-06 Massachusetts Institute Of Technology Delivery of biomolecules to immune cells
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
IL284808B (en) 2015-06-18 2022-07-01 Broad Inst Inc Mutations in the crispr enzyme that reduce unintended effects
US10392674B2 (en) 2015-07-22 2019-08-27 President And Fellows Of Harvard College Evolution of site-specific recombinases
WO2017019895A1 (en) 2015-07-30 2017-02-02 President And Fellows Of Harvard College Evolution of talens
CN116064664A (zh) 2015-10-08 2023-05-05 哈佛学院董事及会员团体 多重基因组编辑
WO2017066781A1 (en) 2015-10-16 2017-04-20 Modernatx, Inc. Mrna cap analogs with modified phosphate linkage
IL310721A (en) 2015-10-23 2024-04-01 Harvard College Nucleobase editors and their uses
US10851369B2 (en) 2016-06-21 2020-12-01 President And Fellows Of Harvard College Frequency-based modulation of diverse species in a nucleic acid library
IL308426A (en) 2016-08-03 2024-01-01 Harvard College Adenosine nuclear base editors and their uses
EP3507364A4 (en) 2016-08-31 2020-05-20 President and Fellows of Harvard College METHODS OF GENERATING NUCLEIC ACID SEQUENCE LIBRARIES FOR IN SITU FLUORESCENT SEQUENCING DETECTION
EP4119663A1 (en) 2016-12-09 2023-01-18 The Broad Institute, Inc. Crispr effector system based diagnostics
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11104937B2 (en) 2017-03-15 2021-08-31 The Broad Institute, Inc. CRISPR effector system based diagnostics
US11021740B2 (en) 2017-03-15 2021-06-01 The Broad Institute, Inc. Devices for CRISPR effector system based diagnostics
CN107939288B (zh) 2017-11-14 2019-04-02 中国科学院地质与地球物理研究所 一种非旋转套的防转装置以及旋转导向装置
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
US20210228627A1 (en) 2018-05-15 2021-07-29 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
CA3105925A1 (en) 2018-07-10 2020-01-16 Alia Therapeutics S.R.L. Vesicles for traceless delivery of guide rna molecules and/or guide rna molecule/rna-guided nuclease complex(es) and a production method thereof
EP3820503A4 (en) 2018-07-13 2022-07-13 Allele Biotechnology & Pharmaceuticals, Inc. METHODS OF ACHIEVING HIGH SPECIFICITY OF GENOMIC EDITING
KR20210049859A (ko) 2018-08-28 2021-05-06 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하는 방법 및 조성물
AU2020242032A1 (en) 2019-03-19 2021-10-07 Massachusetts Institute Of Technology Methods and compositions for editing nucleotide sequences
EP4085141A4 (en) * 2019-12-30 2024-03-06 The Broad Institute, Inc. GENOME EDITING USING ACTIVATED, FULLY ACTIVE CRISPR COMPLEXES OF REVERSE TRANSCRIPTASE
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
MX2023004383A (es) * 2020-10-21 2023-05-04 Massachusetts Inst Technology Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste).

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