AU2017358122B2 - Artificially engineered SC function control system - Google Patents

Artificially engineered SC function control system Download PDF

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Publication number
AU2017358122B2
AU2017358122B2 AU2017358122A AU2017358122A AU2017358122B2 AU 2017358122 B2 AU2017358122 B2 AU 2017358122B2 AU 2017358122 A AU2017358122 A AU 2017358122A AU 2017358122 A AU2017358122 A AU 2017358122A AU 2017358122 B2 AU2017358122 B2 AU 2017358122B2
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nucleic acid
sequence
complementary
gene
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AU2017358122A1 (en
Inventor
Byung Ok Choi
Young Bin Hong
Seokjoong KIM
Jae Young Lee
Jung Min Lee
Dong Woo Song
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Toolgen Inc
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Toolgen Inc
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/315Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type 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)
  • 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)
AU2017358122A 2016-11-14 2017-09-28 Artificially engineered SC function control system Active AU2017358122B2 (en)

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
AU2017358122A1 AU2017358122A1 (en) 2019-07-04
AU2017358122B2 true AU2017358122B2 (en) 2022-12-22

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
AU2017358122A Active AU2017358122B2 (en) 2016-11-14 2017-09-28 Artificially engineered SC function control system

Country Status (9)

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)
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions

Family Cites Families (9)

* 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
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
WO2016021973A1 (ko) 2014-08-06 2016-02-11 주식회사 툴젠 캄필로박터 제주니 crispr/cas 시스템 유래 rgen을 이용한 유전체 교정
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Harrison Pantera ET AL, "A Genome Editing Approach to Studying Pmp22 Enhancer Functionality", The FASEB Journal, (2016-04-01), page 584.2, URL: https://www.fasebj.org/doi/abs/10.1096/fasebj.30.1_supplement.584.2, (2020-04-03) *
JOHN G DOENCH ET AL, "Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9", NATURE BIOTECHNOLOGY, us, (2016-01-18), vol. 34, no. 2, doi:10.1038/nbt.3437, ISSN 1087-0156, pages 184 - 191 *

Also Published As

Publication number Publication date
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
KR20220122592A (ko) 2022-09-02
BR112019009725A2 (pt) 2019-11-19
RU2768043C2 (ru) 2022-03-23
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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