CN110248957B - 经人工操纵的sc功能控制系统 - Google Patents

经人工操纵的sc功能控制系统 Download PDF

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Publication number
CN110248957B
CN110248957B CN201780083263.5A CN201780083263A CN110248957B CN 110248957 B CN110248957 B CN 110248957B CN 201780083263 A CN201780083263 A CN 201780083263A CN 110248957 B CN110248957 B CN 110248957B
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domain
sequence
nucleic acid
complementary
bases
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CN110248957A (zh
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金奭中
宋东佑
洪永彬
崔炳玉
李宰荣
李贞慜
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Toolgen Inc
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Toolgen Inc
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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
    • C12N15/10—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
    • C12N15/113—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
    • 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
    • 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)
  • 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)
  • Toxicology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Ophthalmology & Optometry (AREA)
  • Neurology (AREA)
  • Neurosurgery (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)
CN201780083263.5A 2016-11-14 2017-09-28 经人工操纵的sc功能控制系统 Active CN110248957B (zh)

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

Publications (2)

Publication Number Publication Date
CN110248957A CN110248957A (zh) 2019-09-17
CN110248957B true CN110248957B (zh) 2023-12-19

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CN201780083263.5A Active CN110248957B (zh) 2016-11-14 2017-09-28 经人工操纵的sc功能控制系统

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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)
CA (1) CA3043148A1 (enExample)
RU (2) RU2022105597A (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
KR102670361B1 (ko) * 2017-09-28 2024-05-29 주식회사 툴젠 유전자 발현 조절을 위한 인위적인 게놈 조작
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
WO2020055187A1 (ko) * 2018-09-12 2020-03-19 기초과학연구원 유전자가 변이된 세포의 사멸 유도 조성물 및 상기 조성물을 이용한 유전자가 변형된 세포 사멸 유도 방법
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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
CN105164264A (zh) * 2012-12-12 2015-12-16 布罗德研究所有限公司 用于序列操纵和治疗应用的系统、方法和组合物的递送、工程化和优化

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR100613038B1 (ko) * 2004-07-07 2006-08-16 공주대학교 산학협력단 염색체 17ｐ11.2-ｐ12 지역의 중복을 제외한 유전자돌연변이를 발병 원인으로 하는 ＣＭＴ 질환의 진단방법및 진단키트
CA2690815A1 (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
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
US20160237455A1 (en) * 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
CA2935032C (en) * 2013-12-26 2024-01-23 The General Hospital Corporation Multiplex guide rnas
WO2016021973A1 (ko) * 2014-08-06 2016-02-11 주식회사 툴젠 캄필로박터 제주니 crispr/cas 시스템 유래 rgen을 이용한 유전체 교정
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105164264A (zh) * 2012-12-12 2015-12-16 布罗德研究所有限公司 用于序列操纵和治疗应用的系统、方法和组合物的递送、工程化和优化
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9;DOENCH,J.G. et al;《nature biotechnology》;20160118;摘要及该文章的补充数据材料 *
基因治疗假肥大型肌营养不良症后机体的免疫反应;赵翠萍等;《国际神经病学神经外科学杂志》;20050630(第03期);267-269 *

Also Published As

Publication number Publication date
CA3043148A1 (en) 2018-05-17
JP7338937B2 (ja) 2023-09-05
AU2017358122A1 (en) 2019-07-04
US20190276503A1 (en) 2019-09-12
US12331086B2 (en) 2025-06-17
RU2768043C2 (ru) 2022-03-23
EP3896162B1 (en) 2025-03-05
KR102437228B1 (ko) 2022-08-30
KR20220122592A (ko) 2022-09-02
AU2017358122B2 (en) 2022-12-22
RU2019118283A3 (enExample) 2021-04-26
EP3539980A2 (en) 2019-09-18
EP3896162A1 (en) 2021-10-20
JP2019537446A (ja) 2019-12-26
CN110248957A (zh) 2019-09-17
WO2018088694A2 (ko) 2018-05-17
KR102621539B1 (ko) 2024-01-09
WO2018088694A3 (ko) 2018-08-09
EP3539980A4 (en) 2020-10-07
BR112019009725A2 (pt) 2019-11-19
KR20180054427A (ko) 2018-05-24
RU2019118283A (ru) 2020-12-14
RU2022105597A (ru) 2022-04-04

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