CA3106162A1 - Methods of achieving high specificity of genome editing - Google Patents

Methods of achieving high specificity of genome editing Download PDF

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Publication number
CA3106162A1
CA3106162A1 CA3106162A CA3106162A CA3106162A1 CA 3106162 A1 CA3106162 A1 CA 3106162A1 CA 3106162 A CA3106162 A CA 3106162A CA 3106162 A CA3106162 A CA 3106162A CA 3106162 A1 CA3106162 A1 CA 3106162A1
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Prior art keywords
cas9
mrna
cells
dna
rna
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Application number
CA3106162A
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English (en)
French (fr)
Inventor
Jiwu Wang
Andrew M. Chammas
Alexander Ward
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Allele Biotechnology and Pharmaceuticals Inc
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Allele Biotechnology and Pharmaceuticals Inc
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Publication of CA3106162A1 publication Critical patent/CA3106162A1/en
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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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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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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
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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
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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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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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
    • C12N15/87—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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/16—Aptamers
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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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)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CA3106162A 2018-07-13 2019-07-12 Methods of achieving high specificity of genome editing Pending CA3106162A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862697955P 2018-07-13 2018-07-13
US62/697,955 2018-07-13
PCT/US2019/041551 WO2020014577A1 (en) 2018-07-13 2019-07-12 Methods of achieving high specificity of genome editing

Publications (1)

Publication Number Publication Date
CA3106162A1 true CA3106162A1 (en) 2020-01-16

Family

ID=69141767

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3106162A Pending CA3106162A1 (en) 2018-07-13 2019-07-12 Methods of achieving high specificity of genome editing

Country Status (7)

Country Link
US (1) US20220195403A1 (https=)
EP (1) EP3820503A4 (https=)
JP (2) JP7590952B2 (https=)
KR (1) KR20210031482A (https=)
CA (1) CA3106162A1 (https=)
TW (1) TW202023605A (https=)
WO (1) WO2020014577A1 (https=)

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EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
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
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
KR20240099393A (ko) 2021-11-01 2024-06-28 톰 바이오사이언시스, 인코포레이티드 유전자 편집 기구와 핵산 카고의 동시 전달을 위한 단일 작제물 플랫폼
WO2023122764A1 (en) 2021-12-22 2023-06-29 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
TW202346588A (zh) * 2022-03-04 2023-12-01 大陸商益杰立科（上海）生物科技有限公司 基因組編輯的組成物和方法
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
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
CN115312122B (zh) * 2022-10-12 2022-12-16 之江实验室 一种CRISPR-Cas酶可突变位点推荐方法和装置
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
IL324634A (en) 2023-05-15 2026-01-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of hbv gene expression
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
EP4677108A1 (en) 2024-04-22 2026-01-14 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo

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US9862926B2 (en) * 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
EP3011030B1 (en) * 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
SMT202100691T1 (it) * 2013-07-11 2022-01-10 Modernatx Inc Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso
US9340799B2 (en) * 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US10900034B2 (en) * 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
EP3233880A1 (en) * 2014-12-16 2017-10-25 Novartis AG End capped nucleic acid molecules
CN104611368B (zh) * 2015-01-15 2018-05-25 中国科学院广州生物医药与健康研究院 重组后不产生移码突变的载体、在爪蛙基因组中进行基因定点敲入的方法及应用
US20180112213A1 (en) * 2015-03-25 2018-04-26 Editas Medicine, Inc. Crispr/cas-related methods, compositions and components
US10188750B1 (en) * 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
WO2017160662A1 (en) * 2016-03-12 2017-09-21 The Regents Of The University Of California Biodegradable vectors for efficient rna delivery
EP4286523A3 (en) * 2016-12-20 2024-03-20 Bristol-Myers Squibb Company Methods for increasing the efficiency of homology directed repair (hdr) in the cellular genome

Also Published As

Publication number Publication date
EP3820503A1 (en) 2021-05-19
JP7590952B2 (ja) 2024-11-27
JP2021530988A (ja) 2021-11-18
TW202023605A (zh) 2020-07-01
WO2020014577A1 (en) 2020-01-16
KR20210031482A (ko) 2021-03-19
EP3820503A4 (en) 2022-07-13
JP2024164090A (ja) 2024-11-26
US20220195403A1 (en) 2022-06-23

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