CA3043148A1 - Artificially engineered sc function control system - Google Patents

Artificially engineered sc function control system Download PDF

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Publication number
CA3043148A1
CA3043148A1 CA3043148A CA3043148A CA3043148A1 CA 3043148 A1 CA3043148 A1 CA 3043148A1 CA 3043148 A CA3043148 A CA 3043148A CA 3043148 A CA3043148 A CA 3043148A CA 3043148 A1 CA3043148 A1 CA 3043148A1
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Canada
Prior art keywords
nucleic acid
domain
gene
sequence
function
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Pending
Application number
CA3043148A
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English (en)
French (fr)
Inventor
Seokjoong KIM
Dong Woo Song
Young Bin Hong
Byung Ok Choi
Jae Young Lee
Jung Min Lee
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Toolgen Inc
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Toolgen Inc
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Publication of CA3043148A1 publication Critical patent/CA3043148A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (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)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Neurosurgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Neurology (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)
CA3043148A 2016-11-14 2017-09-28 Artificially engineered sc function control system Pending CA3043148A1 (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 (1)

Publication Number Publication Date
CA3043148A1 true CA3043148A1 (en) 2018-05-17

Family

ID=62109586

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3043148A Pending CA3043148A1 (en) 2016-11-14 2017-09-28 Artificially engineered sc function control system

Country Status (10)

Country Link
US (1) US12331086B2 (OSRAM)
EP (2) EP3539980A4 (OSRAM)
JP (1) JP7338937B2 (OSRAM)
KR (2) KR102437228B1 (OSRAM)
CN (1) CN110248957B (OSRAM)
AU (1) AU2017358122B2 (OSRAM)
BR (1) BR112019009725A2 (OSRAM)
CA (1) CA3043148A1 (OSRAM)
RU (2) RU2022105597A (OSRAM)
WO (1) WO2018088694A2 (OSRAM)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220002721A1 (en) * 2018-09-25 2022-01-06 Centre National De La Recherche Scientifique Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease

Families Citing this family (7)

* 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
BR112020006428A2 (pt) * 2017-09-28 2020-09-24 Toolgen Incorporated manipulação de genoma artificial para regulação de expressão de genes
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
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

Family Cites Families (10)

* 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
WO2008154644A1 (en) 2007-06-12 2008-12-18 Case Western Reserve University Targeted cell death
SG11201503059XA (en) 2012-10-23 2015-06-29 Toolgen Inc 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
BR112015013784A2 (pt) * 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
JP6625971B2 (ja) * 2013-06-17 2019-12-25 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化
US20160237455A1 (en) * 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
AU2014370416B2 (en) * 2013-12-26 2021-03-11 The General Hospital Corporation Multiplex guide RNAs
EP3152319A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
AU2015299850B2 (en) * 2014-08-06 2020-08-13 Institute For Basic Science Genome editing using Campylobacter jejuni CRISPR/CAS system-derived RGEN
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220002721A1 (en) * 2018-09-25 2022-01-06 Centre National De La Recherche Scientifique Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease
US11939577B2 (en) * 2018-09-25 2024-03-26 Centre National De La Recherche Scientifique Antisense RNA targeting PMP22 for the treatment of Charcot-Marie-Tooth 1A disease
AU2019350357B2 (en) * 2018-09-25 2025-10-16 Centre National De La Recherche Scientifique Antisense RNA targeting PMP22 for the treatment of Charcot-Marie-Tooth 1A disease

Also Published As

Publication number Publication date
RU2022105597A (ru) 2022-04-04
BR112019009725A2 (pt) 2019-11-19
AU2017358122A1 (en) 2019-07-04
RU2019118283A3 (OSRAM) 2021-04-26
KR102621539B1 (ko) 2024-01-09
US12331086B2 (en) 2025-06-17
RU2768043C2 (ru) 2022-03-23
JP7338937B2 (ja) 2023-09-05
AU2017358122B2 (en) 2022-12-22
KR102437228B1 (ko) 2022-08-30
KR20220122592A (ko) 2022-09-02
KR20180054427A (ko) 2018-05-24
WO2018088694A3 (ko) 2018-08-09
EP3539980A2 (en) 2019-09-18
EP3539980A4 (en) 2020-10-07
EP3896162A1 (en) 2021-10-20
CN110248957B (zh) 2023-12-19
RU2019118283A (ru) 2020-12-14
EP3896162B1 (en) 2025-03-05
CN110248957A (zh) 2019-09-17
WO2018088694A2 (ko) 2018-05-17
US20190276503A1 (en) 2019-09-12
JP2019537446A (ja) 2019-12-26

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