RU2768043C2 - Искусственно созданная система управления функцией шк - Google Patents

Искусственно созданная система управления функцией шк Download PDF

Info

Publication number
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
Authority
RU
Russia
Prior art keywords
domain
sequence
complementary
nucleic acid
target
Prior art date
Application number
RU2019118283A
Other languages
English (en)
Russian (ru)
Other versions
RU2019118283A (ru
RU2019118283A3 (enExample
Inventor
Сеок Дзоонг КИМ
Донг Воо СОНГ
Йонг Бин ХОНГ
Биунг Ок ЧОИ
Дзае Йоунг ЛИ
Дзунг Мин ЛИ
Original Assignee
Тулджен Инкорпорейтед
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 Тулджен Инкорпорейтед filed Critical Тулджен Инкорпорейтед
Publication of RU2019118283A publication Critical patent/RU2019118283A/ru
Publication of RU2019118283A3 publication Critical patent/RU2019118283A3/ru
Application granted granted Critical
Publication of RU2768043C2 publication Critical patent/RU2768043C2/ru

Links

Images

Classifications

    • 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]

Landscapes

  • 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)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • 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)
  • Public Health (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (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)

Application Number Title Priority Date Filing Date
RU2022105597A Division RU2022105597A (ru) 2016-11-14 2017-09-28 Искусственно созданная система управления функцией шк

Publications (3)

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

Family

ID=62109586

Family Applications (2)

Application Number Title Priority Date Filing Date
RU2019118283A RU2768043C2 (ru) 2016-11-14 2017-09-28 Искусственно созданная система управления функцией шк
RU2022105597A RU2022105597A (ru) 2016-11-14 2017-09-28 Искусственно созданная система управления функцией шк

Family Applications After (1)

Application Number Title Priority Date Filing Date
RU2022105597A RU2022105597A (ru) 2016-11-14 2017-09-28 Искусственно созданная система управления функцией шк

Country Status (10)

Country Link
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)
CA (1) CA3043148A1 (enExample)
RU (2) RU2768043C2 (enExample)
WO (1) WO2018088694A2 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
SG11202002130WA (en) * 2017-09-28 2020-04-29 Toolgen Inc Artificial genome manipulation for gene expression regulation
CA3111477A1 (en) * 2018-09-12 2020-03-19 Institute For Basic Science Composition for inducing death of cells having mutated gene, and method for inducing death of cells having mutated gene by using composition
EP3628735A1 (en) * 2018-09-25 2020-04-01 Centre National De La Recherche Scientifique Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease
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)

* Cited by examiner, † Cited by third party
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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008154644A1 (en) * 2007-06-12 2008-12-18 Case Western Reserve University Targeted cell death
KR101656236B1 (ko) * 2012-10-23 2016-09-12 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
AU2013359199C1 (en) 2012-12-12 2021-06-17 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2015099850A1 (en) 2013-12-26 2015-07-02 The General Hospital Corporation Multiplex guide rnas
AU2014281026B2 (en) 2013-06-17 2020-05-28 Massachusetts Institute Of Technology Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3151846A4 (en) * 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
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

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
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. *

Also Published As

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

Similar Documents

Publication Publication Date Title
RU2768043C2 (ru) Искусственно созданная система управления функцией шк
JP7440043B2 (ja) 遺伝子発現調節のための人為的なゲノム操作
EP3690045A2 (en) Gene manipulation for treatment of retinal dysfunction disorder
Liu et al. Allele-specific gene-editing approach for vision loss restoration in RHO-associated retinitis pigmentosa
US20200101173A1 (en) Genome Editing System For Repeat Expansion Mutation
US12163149B2 (en) Artificial genome manipulation for gene expression regulation
KR102847166B1 (ko) 항응고 인자들의 유전자 에디팅
HK40014170B (zh) 经人工操纵的sc功能控制系统
EP4342988A1 (en) Compositions and methods for modification of the expression of a snca gene
HK40014170A (en) Artificially engineered sc function control system
WO2025227157A1 (en) Modified cells and uses thereof
HK40035616B (zh) 用於基因表逹调节的人工基因组操纵
HK40035616A (en) Artificial genome manipultation for gene expression regulation