RU2768043C2 - Искусственно созданная система управления функцией шк - Google Patents

Искусственно созданная система управления функцией шк Download PDF

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RU2768043C2
RU2768043C2 RU2019118283A RU2019118283A RU2768043C2 RU 2768043 C2 RU2768043 C2 RU 2768043C2 RU 2019118283 A RU2019118283 A RU 2019118283A RU 2019118283 A RU2019118283 A RU 2019118283A RU 2768043 C2 RU2768043 C2 RU 2768043C2
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sequence
complementary
nucleic acid
target
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RU2019118283A
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RU2019118283A3 (enExample
RU2019118283A (ru
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Сеок Дзоонг КИМ
Донг Воо СОНГ
Йонг Бин ХОНГ
Биунг Ок ЧОИ
Дзае Йоунг ЛИ
Дзунг Мин ЛИ
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Тулджен Инкорпорейтед
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/315—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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]
    • 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
    • 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]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Ophthalmology & Optometry (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)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
RU2019118283A 2016-11-14 2017-09-28 Искусственно созданная система управления функцией шк RU2768043C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662421516P 2016-11-14 2016-11-14
US62/421,516 2016-11-14
PCT/KR2017/010897 WO2018088694A2 (ko) 2016-11-14 2017-09-28 인위적으로 조작된 sc 기능 조절 시스템

Related Child Applications (1)

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RU2022105597A Division RU2022105597A (ru) 2016-11-14 2017-09-28 Искусственно созданная система управления функцией шк

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RU2019118283A RU2019118283A (ru) 2020-12-14
RU2019118283A3 RU2019118283A3 (enExample) 2021-04-26
RU2768043C2 true RU2768043C2 (ru) 2022-03-23

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RU2022105597A RU2022105597A (ru) 2016-11-14 2017-09-28 Искусственно созданная система управления функцией шк

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US (1) US12331086B2 (enExample)
EP (2) EP3539980A4 (enExample)
JP (1) JP7338937B2 (enExample)
KR (2) KR102437228B1 (enExample)
CN (1) CN110248957B (enExample)
AU (1) AU2017358122B2 (enExample)
BR (1) BR112019009725A2 (enExample)
RU (2) RU2768043C2 (enExample)
WO (1) WO2018088694A2 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2022103641A (ru) * 2017-09-28 2022-03-18 Тулджен Инкорпорейтед Искусственная модификация генома для регуляции экспрессии гена
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
JP7410154B2 (ja) * 2018-09-12 2024-01-09 インスティチュート フォー ベーシック サイエンス 遺伝的に変異された細胞の死滅誘導組成物及び該組成物を用いた遺伝的に変異された細胞の死滅誘導方法
EP3798301A1 (en) * 2019-09-25 2021-03-31 KWS SAAT SE & Co. KGaA Promoter repression
WO2021184021A1 (en) * 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting pmp22
CN114621922A (zh) * 2022-02-15 2022-06-14 中山大学附属第五医院 一种大鼠海绵体神经来源施旺细胞的提取纯化和培养方法
WO2025079278A1 (ja) * 2023-10-10 2025-04-17 国立大学法人 東京医科歯科大学 シャルコー・マリー・トゥース病の処置剤

Citations (3)

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KR20060003673A (ko) * 2004-07-07 2006-01-11 공주대학교 산학협력단 염색체 17ｐ11.2-ｐ12 지역의 중복을 제외한 유전자돌연변이를 발병 원인으로 하는 ＣＭＴ 질환의 진단방법및 진단키트
WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
WO2016021973A1 (ko) * 2014-08-06 2016-02-11 주식회사 툴젠 캄필로박터 제주니 crispr/cas 시스템 유래 rgen을 이용한 유전체 교정

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* Cited by examiner, † Cited by third party
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US8124749B2 (en) * 2007-06-12 2012-02-28 Case Western Reserve University Targeted cell death
EP2912175B1 (en) 2012-10-23 2018-08-22 Toolgen Incorporated 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
WO2014093622A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP3674411A1 (en) * 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3090044B1 (en) * 2013-12-26 2021-11-03 The General Hospital Corporation Multiplex guide rnas
EP3152319A4 (en) * 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
KR20060003673A (ko) * 2004-07-07 2006-01-11 공주대학교 산학협력단 염색체 17ｐ11.2-ｐ12 지역의 중복을 제외한 유전자돌연변이를 발병 원인으로 하는 ＣＭＴ 질환의 진단방법및 진단키트
WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
WO2016021973A1 (ko) * 2014-08-06 2016-02-11 주식회사 툴젠 캄필로박터 제주니 crispr/cas 시스템 유래 rgen을 이용한 유전체 교정

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JUN LI et al., The PMP22 Gene and Its Related Diseases, Mol Neurobiol, 2013, Vol.47, pp.673-698. *
JUN LI et al., The PMP22 Gene and Its Related Diseases, Mol Neurobiol, 2013, Vol.47, pp.673-698. WO2016021973 A1. АВДЕЕВА Ж.И. и др., Требования к производству и контролю препаратов на основе моноклональных антител, применяемых для лечения, Биопрепараты, 2010, с.11-14. *
АВДЕЕВА Ж.И. и др., Требования к производству и контролю препаратов на основе моноклональных антител, применяемых для лечения, Биопрепараты, 2010, с.11-14. *

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RU2019118283A3 (enExample) 2021-04-26
KR102437228B1 (ko) 2022-08-30
CA3043148A1 (en) 2018-05-17
US20190276503A1 (en) 2019-09-12
JP2019537446A (ja) 2019-12-26
KR102621539B1 (ko) 2024-01-09
EP3896162B1 (en) 2025-03-05
US12331086B2 (en) 2025-06-17
AU2017358122B2 (en) 2022-12-22
KR20220122592A (ko) 2022-09-02
BR112019009725A2 (pt) 2019-11-19
WO2018088694A3 (ko) 2018-08-09
RU2019118283A (ru) 2020-12-14
EP3896162A1 (en) 2021-10-20
KR20180054427A (ko) 2018-05-24
RU2022105597A (ru) 2022-04-04
CN110248957A (zh) 2019-09-17
WO2018088694A2 (ko) 2018-05-17
EP3539980A2 (en) 2019-09-18
EP3539980A4 (en) 2020-10-07
JP7338937B2 (ja) 2023-09-05
CN110248957B (zh) 2023-12-19
AU2017358122A1 (en) 2019-07-04

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