JP7338937B2 - 人工的に操作されたｓｃ機能制御システム - Google Patents

人工的に操作されたｓｃ機能制御システム Download PDF

Info

Publication number
JP7338937B2
JP7338937B2 JP2019524226A JP2019524226A JP7338937B2 JP 7338937 B2 JP7338937 B2 JP 7338937B2 JP 2019524226 A JP2019524226 A JP 2019524226A JP 2019524226 A JP2019524226 A JP 2019524226A JP 7338937 B2 JP7338937 B2 JP 7338937B2
Authority
JP
Japan
Prior art keywords
domain
nucleic acid
sequence
complementary
target
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2019524226A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019537446A (ja
JP2019537446A5 (enExample
Inventor
キム、ソクジョン
ウー ソン、ドン
ビン ホン、ヨン
オク チョイ、ビュン
ヨン リー、ジェ
ミン リー、ジュン
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Toolgen Inc
Original Assignee
Toolgen Inc
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 Toolgen Inc filed Critical Toolgen Inc
Publication of JP2019537446A publication Critical patent/JP2019537446A/ja
Publication of JP2019537446A5 publication Critical patent/JP2019537446A5/ja
Application granted granted Critical
Publication of JP7338937B2 publication Critical patent/JP7338937B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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]

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)
JP2019524226A 2016-11-14 2017-09-28 人工的に操作されたｓｃ機能制御システム Active JP7338937B2 (ja)

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 (3)

Publication Number Publication Date
JP2019537446A JP2019537446A (ja) 2019-12-26
JP2019537446A5 JP2019537446A5 (enExample) 2020-11-12
JP7338937B2 true JP7338937B2 (ja) 2023-09-05

Family

ID=62109586

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019524226A Active JP7338937B2 (ja) 2016-11-14 2017-09-28 人工的に操作されたｓｃ機能制御システム

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 (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
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
KR101817482B1 (ko) * 2014-08-06 2018-02-22 주식회사 툴젠 캄필로박터 제주니 crispr/cas 시스템 유래 rgen을 이용한 유전체 교정
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof

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 (3)

* Cited by examiner, † Cited by third party
Title
Journal of Integrative Plant Biology，2016年08月，Vol. 58，p. 705-712
Nature Biotech.，2016年02月，Vol. 34，p. 184-191
Shijie Huaren Xiaohua Zashi，2015年，Vol. 23，p. 3742-3748

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
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
RU2768043C2 (ru) 2022-03-23
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
JP7338937B2 (ja) 人工的に操作されたｓｃ機能制御システム
JP7440043B2 (ja) 遺伝子発現調節のための人為的なゲノム操作
JP2020527030A (ja) 肝臓において目的のタンパク質を発現するためのプラットフォーム
JP2024038021A (ja) 機能的ミエリン産生を増進させるための方法および組成物
JP2024531217A (ja) 遺伝子改変高忠実度ｏｍｎｉ－５０ヌクレアーゼバリアント
JP2020528735A (ja) 反復伸長変異のためのゲノム編集システム
KR102847166B1 (ko) 항응고 인자들의 유전자 에디팅
US12163149B2 (en) Artificial genome manipulation for gene expression regulation
JP2024542883A (ja) 改変された忠実度の高いｏｍｎｉ－７９ヌクレアーゼバリアント
HK40014170B (zh) 经人工操纵的sc功能控制系统
HK40014170A (en) Artificially engineered sc function control system
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

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200928

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200928

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20211109

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220209

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220705

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221004

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230131

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230420

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230725

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230821

R150 Certificate of patent or registration of utility model

Ref document number: 7338937

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150