MX2023004383A - Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste). - Google Patents

Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste).

Info

Publication number
MX2023004383A
MX2023004383A MX2023004383A MX2023004383A MX2023004383A MX 2023004383 A MX2023004383 A MX 2023004383A MX 2023004383 A MX2023004383 A MX 2023004383A MX 2023004383 A MX2023004383 A MX 2023004383A MX 2023004383 A MX2023004383 A MX 2023004383A
Authority
MX
Mexico
Prior art keywords
site
paste
specific
compositions
methods
Prior art date
Application number
MX2023004383A
Other languages
English (en)
Inventor
Jonathan Gootenberg
Omar Abudayyeh
Original Assignee
Massachusetts Inst Technology
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 Massachusetts Inst Technology filed Critical Massachusetts Inst Technology
Publication of MX2023004383A publication Critical patent/MX2023004383A/es

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
    • C12N15/10—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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal 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
    • C12N15/87—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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • 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
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Abstract

La presente descripción proporciona sistemas, métodos y composiciones para la ingeniería genética específica del sitio usando la adición programable a través de elementos objetivo específicos del sitio (PASTE). PASTE comprende la adición de un sitio de integración en un genoma objetivo seguida por la inserción de uno o más genes de interés o una o más secuencias de ácidos nucleicos de interés en el sitio. PASTE combina las tecnologías de edición de genes y las tecnologías de integrasa para lograr la incorporación unidireccional de genes en un genoma para el tratamiento y diagnóstico de enfermedades.
MX2023004383A 2020-10-21 2021-10-21 Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste). MX2023004383A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063094803P 2020-10-21 2020-10-21
US202163222550P 2021-07-16 2021-07-16
PCT/US2021/056006 WO2022087235A1 (en) 2020-10-21 2021-10-21 Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)

Publications (1)

Publication Number Publication Date
MX2023004383A true MX2023004383A (es) 2023-05-04

Family

ID=78821040

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023004383A MX2023004383A (es) 2020-10-21 2021-10-21 Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste).

Country Status (10)

Country Link
US (6) US20220145293A1 (es)
EP (1) EP4232583A1 (es)
JP (1) JP2023546597A (es)
KR (1) KR20230091894A (es)
CN (1) CN116419975A (es)
AU (1) AU2021364781A1 (es)
CA (1) CA3196116A1 (es)
IL (1) IL301368A (es)
MX (1) MX2023004383A (es)
WO (1) WO2022087235A1 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220145293A1 (en) 2020-10-21 2022-05-12 Massachusetts Institute Of Technology Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
GB202113933D0 (en) * 2021-09-29 2021-11-10 Genome Res Ltd Methods for gene editing
WO2023076898A1 (en) * 2021-10-25 2023-05-04 The Broad Institute, Inc. Methods and compositions for editing a genome with prime editing and a recombinase
WO2023077148A1 (en) * 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2023114992A1 (en) * 2021-12-17 2023-06-22 Massachusetts Institute Of Technology Programmable insertion approaches via reverse transcriptase recruitment
WO2023122764A1 (en) 2021-12-22 2023-06-29 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
WO2023205708A1 (en) 2022-04-20 2023-10-26 Massachusetts Institute Of Technology SITE SPECIFIC GENETIC ENGINEERING UTILIZING TRANS-TEMPLATE RNAs
US20230407280A1 (en) 2022-04-20 2023-12-21 Massachusetts Institute Of Technology Programmable gene editing using guide rna pair
WO2023205744A1 (en) * 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023215831A1 (en) * 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023225670A2 (en) * 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024020346A2 (en) 2022-07-18 2024-01-25 Renagade Therapeutics Management Inc. Gene editing components, systems, and methods of use
WO2024020587A2 (en) * 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
EA038924B1 (ru) 2012-05-25 2021-11-10 Те Риджентс Оф Те Юниверсити Оф Калифорния Способы и композиции рнк-специфической модификации днк-мишени и рнк-специфической модуляции транскрипции
ES2576128T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones para la manipulación de secuencias con dominios funcionales
RU2766685C2 (ru) 2012-12-17 2022-03-15 Президент Энд Фэллоуз Оф Харвард Коллидж Рнк-направляемая инженерия генома человека
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US20140349400A1 (en) * 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US9322037B2 (en) * 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
CA2952613A1 (en) 2014-06-17 2015-12-23 Poseida Therapeutics, Inc. A method for directing proteins to specific loci in the genome and uses thereof
US10059940B2 (en) 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
WO2017151719A1 (en) 2016-03-01 2017-09-08 University Of Florida Research Foundation, Incorporated Molecular cell diary system
WO2017180711A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Grna fusion molecules, gene editing systems, and methods of use thereof
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
WO2018049168A1 (en) 2016-09-09 2018-03-15 The Board Of Trustees Of The Leland Stanford Junior University High-throughput precision genome editing
WO2018049175A1 (en) 2016-09-12 2018-03-15 Mems Drive, Inc. Mems actuation systems and methods
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
CA3084825A1 (en) 2017-12-14 2019-06-20 Crispr Therapeutics Ag Novel rna-programmable endonuclease systems and their use in genome editing and other applications
EP3844272A1 (en) 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
MX2021011426A (es) * 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
CN113939593A (zh) 2019-06-06 2022-01-14 因思科瑞普特公司 用于递归的核酸指导的细胞编辑的处治
CN114981409A (zh) 2019-09-03 2022-08-30 美洛德生物医药公司 用于基因组整合的方法和组合物
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
WO2021138469A1 (en) * 2019-12-30 2021-07-08 The Broad Institute, Inc. Genome editing using reverse transcriptase enabled and fully active crispr complexes
WO2021188840A1 (en) 2020-03-19 2021-09-23 Rewrite Therapeutics, Inc. Methods and compositions for directed genome editing
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
US11299731B1 (en) * 2020-09-15 2022-04-12 Inscripta, Inc. CRISPR editing to embed nucleic acid landing pads into genomes of live cells
JP2023543803A (ja) 2020-09-24 2023-10-18 ザ ブロード インスティテュート，インコーポレーテッド プライム編集ガイドｒｎａ、その組成物、及びその使用方法
US20220145293A1 (en) 2020-10-21 2022-05-12 Massachusetts Institute Of Technology Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
EP4240840A1 (en) 2020-11-05 2023-09-13 University of Washington Precise genome deletion and replacement method based on prime editing

Also Published As

Publication number Publication date
US11952571B2 (en) 2024-04-09
US20240076662A1 (en) 2024-03-07
IL301368A (en) 2023-05-01
US20230279391A1 (en) 2023-09-07
AU2021364781A1 (en) 2023-06-01
US20220154224A1 (en) 2022-05-19
WO2022087235A1 (en) 2022-04-28
JP2023546597A (ja) 2023-11-06
EP4232583A1 (en) 2023-08-30
US11834658B2 (en) 2023-12-05
US20230135673A1 (en) 2023-05-04
CA3196116A1 (en) 2022-04-28
US11572556B2 (en) 2023-02-07
US11827881B2 (en) 2023-11-28
CN116419975A (zh) 2023-07-11
US20220145293A1 (en) 2022-05-12
KR20230091894A (ko) 2023-06-23
US20240067961A1 (en) 2024-02-29

Similar Documents

Publication Publication Date Title
MX2023004383A (es) Sistemas, metodos y composiciones para la ingenieria genetica especifica del sitio usando la adicion programable a traves de elementos objetivo especificos del sitio (paste).
JP7083595B2 (ja) 高反復モチーフを備えたｄｎａ配列を効率的かつ特異的に標的とするレアカットエンドヌクレアーゼの設計
KR101785847B1 (ko) 선형 이중가닥 ＤＮＡ를 활용한 ＣＲＩＳＰＲ／Ｃａｓ９ 시스템을 이용한 표적 유전체 교정
US11535871B2 (en) Optimized gene editing utilizing a recombinant endonuclease system
HRP20200523T1 (hr) Sastavljanje dnk posredovano nukleazom
JOP20210231A1 (ar) مركبات ترابطية مستهدفة كمركبات علاجية
WO2017053431A3 (en) Allele selective gene editing and uses thereof
US20170314016A1 (en) Method for rgen rnp delivery using 5'-phosphate removed rna
JP2016537028A5 (es)
WO2019103442A3 (ko) CRISPR/Cpf1 시스템을 이용한 유전체 편집용 조성물 및 이의 용도
RU2020104969A (ru) Способы и продукты для получения и доставки нуклеиновых кислот
CN107429263A (zh) 调控基因组编辑的方法
JP2021500889A5 (es)
IL274179B2 (en) Targeted replacement of endogenous T cell receptors
Ousterout et al. The development of TALE nucleases for biotechnology
RU2015120524A (ru) Способы и продукты для экспрессии белков в клетках
WO2007014275A3 (en) Targeted integration and expression of exogenous nucleic acid sequences
EA202091673A1 (ru) Способ амплификации нуклеиновых кислот
EP4245861A3 (en) Single tube bead-based dna co-barcoding for accurate and cost-effective sequencing, haplotyping, and assembly
WO2011142798A3 (en) Methods of preparing targeted aptamer prodrugs
JP2024515936A (ja) Ｃａｓ酵素を用いたマルチプレックス編集
Reyes et al. Time matters: gene editing at the mouse 2-cell embryo stage boosts Knockin efficiency
Rahim et al. Systematic review on the comparison of molecular gene editing tools
Pacesa et al. Past, present, and future of CRISPR genome editing technologies
BR112019019673A2 (pt) método e agente para alterar um sítio alvejado de um dna em uma célula, complexo, e, ácido nucléico