BR112020009268A2 - plataformas de distribuição de crispr direcionadas - Google Patents

plataformas de distribuição de crispr direcionadas Download PDF

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Publication number
BR112020009268A2
BR112020009268A2 BR112020009268-1A BR112020009268A BR112020009268A2 BR 112020009268 A2 BR112020009268 A2 BR 112020009268A2 BR 112020009268 A BR112020009268 A BR 112020009268A BR 112020009268 A2 BR112020009268 A2 BR 112020009268A2
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BR
Brazil
Prior art keywords
nucleotides
sgrna
sequence
fact
cas9
Prior art date
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BR112020009268-1A
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English (en)
Portuguese (pt)
Inventor
Erik Joseph Sontheimer
Raed Ibraheim
Wen Xue
Aamir Mir
Alireza Edraki
Gainetdinov Ildar
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University Of Massachusetts
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Publication of BR112020009268A2 publication Critical patent/BR112020009268A2/pt

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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
    • C12N15/63—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
    • C12N15/86—Viral vectors
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Manufacturing & Machinery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
BR112020009268-1A 2017-11-10 2018-11-09 plataformas de distribuição de crispr direcionadas BR112020009268A2 (pt)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762584310P 2017-11-10 2017-11-10
US62/584,310 2017-11-10
US201762596375P 2017-12-08 2017-12-08
US62/596,375 2017-12-08
US201862667084P 2018-05-04 2018-05-04
US62/667,084 2018-05-04
PCT/US2018/060126 WO2019094791A2 (en) 2017-11-10 2018-11-09 Targeted crispr delivery platforms

Publications (1)

Publication Number Publication Date
BR112020009268A2 true BR112020009268A2 (pt) 2020-11-17

Family

ID=66438139

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112020009268-1A BR112020009268A2 (pt) 2017-11-10 2018-11-09 plataformas de distribuição de crispr direcionadas

Country Status (13)

Country Link
US (3) US20220389447A9 (enExample)
EP (1) EP3707254A4 (enExample)
JP (2) JP2021502097A (enExample)
KR (1) KR20200080314A (enExample)
CN (1) CN111868240A (enExample)
AU (2) AU2018364993B2 (enExample)
BR (1) BR112020009268A2 (enExample)
CA (1) CA3082370A1 (enExample)
CO (1) CO2020007046A2 (enExample)
IL (2) IL274526B2 (enExample)
MX (1) MX2020004777A (enExample)
SG (1) SG11202005103RA (enExample)
WO (1) WO2019094791A2 (enExample)

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Publication number Priority date Publication date Assignee Title
WO2020067004A1 (ja) * 2018-09-25 2020-04-02 公益財団法人微生物化学研究会 新規ウイルスベクターおよびその製造方法と使用方法
KR102834042B1 (ko) 2019-02-05 2025-07-18 파라그라프 유에스에이 인코포레이티드 효율적인 표적화된 게놈 조작제의 향상된 선택을 위한 기기, 방법, 컴퓨터 프로그램 제품 및 시스템
CN113728097A (zh) * 2019-02-14 2021-11-30 宏基因组学知识产权技术有限责任公司 具有ruvc结构域的酶
CN110257406B (zh) * 2019-06-21 2022-07-22 安徽省农业科学院水稻研究所 密码子植物化改造的Plant Nme2Cas9基因及其应用
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
WO2021122944A1 (en) 2019-12-18 2021-06-24 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
CN113652411B (zh) * 2021-07-30 2025-01-10 复旦大学 Cas9蛋白、含有Cas9蛋白的基因编辑系统及应用
AU2022335499A1 (en) 2021-08-27 2024-02-22 Metagenomi, Inc. Enzymes with ruvc domains
WO2023064813A2 (en) * 2021-10-13 2023-04-20 University Of Massachusetts Modified guide rnas for neisseria meningitidis cas9
IL312508A (en) 2021-11-03 2024-07-01 Intellia Therapeutics Inc Polynucleotides, compounds and methods for genome editing
AU2022381173A1 (en) * 2021-11-03 2024-05-02 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
TW202426646A (zh) 2022-12-21 2024-07-01 美商英特利亞醫療公司 用於前蛋白轉化酶枯草桿菌蛋白酶ｋｅｘｉｎ９（ｐｃｓｋ９）編輯之組合物及方法
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
TW202515992A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對轉形生長因子β受體２型（ＴＧＦβＲ２）進行基因修飾之組合物及方法
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy

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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
EP3825406A1 (en) * 2013-06-17 2021-05-26 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
EP3666892A1 (en) * 2013-07-10 2020-06-17 President and Fellows of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
AU2014350051A1 (en) * 2013-11-18 2016-07-07 Crispr Therapeutics Ag CRISPR-Cas system materials and methods
JP2016538001A (ja) * 2013-11-28 2016-12-08 ホライズン・ジェノミクス・ゲーエムベーハー 体細胞半数体ヒト細胞株
EP4219699A1 (en) * 2013-12-12 2023-08-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
EP3080271B1 (en) * 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
WO2016049258A2 (en) * 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
US10190106B2 (en) * 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
WO2016179038A1 (en) * 2015-05-01 2016-11-10 Spark Therapeutics, Inc. ADENO-ASSOCIATED VIRUS-MEDIATED CRISPR-Cas9 TREATMENT OF OCULAR DISEASE
US11732258B2 (en) * 2016-11-02 2023-08-22 President And Fellows Of Harvard College Engineered guide RNA sequences for in situ detection and sequencing
KR20200017479A (ko) * 2017-07-31 2020-02-18 시그마-알드리치 컴퍼니., 엘엘씨 Crispr/cas 활성제 시스템용 합성 유도 rna

Also Published As

Publication number Publication date
US20250223611A1 (en) 2025-07-10
SG11202005103RA (en) 2020-06-29
AU2023200084B2 (en) 2025-10-16
AU2018364993B2 (en) 2022-10-06
IL274526B1 (en) 2025-01-01
JP2021502097A (ja) 2021-01-28
US20220389447A9 (en) 2022-12-08
AU2023200084A1 (en) 2023-02-09
MX2020004777A (es) 2020-10-08
WO2019094791A2 (en) 2019-05-16
WO2019094791A3 (en) 2019-06-20
EP3707254A4 (en) 2021-08-18
JP2024019727A (ja) 2024-02-09
CA3082370A1 (en) 2019-05-16
IL317505A (en) 2025-02-01
US20250197889A1 (en) 2025-06-19
US20190338308A1 (en) 2019-11-07
KR20200080314A (ko) 2020-07-06
AU2018364993A1 (en) 2020-06-11
CO2020007046A2 (es) 2020-08-31
IL274526B2 (en) 2025-05-01
EP3707254A2 (en) 2020-09-16
CN111868240A (zh) 2020-10-30
IL274526A (en) 2020-06-30

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