KR102612148B1 - 재조합 인간 II형 미토콘드리아 다이네인-유사 GTPase의 유전자 서열 및 이의 용도 - Google Patents

재조합 인간 II형 미토콘드리아 다이네인-유사 GTPase의 유전자 서열 및 이의 용도 Download PDF

Info

Publication number
KR102612148B1
KR102612148B1 KR1020217008407A KR20217008407A KR102612148B1 KR 102612148 B1 KR102612148 B1 KR 102612148B1 KR 1020217008407 A KR1020217008407 A KR 1020217008407A KR 20217008407 A KR20217008407 A KR 20217008407A KR 102612148 B1 KR102612148 B1 KR 102612148B1
Authority
KR
South Korea
Prior art keywords
nucleic acid
sequence
vector
gtpase
recombinant nucleic
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
KR1020217008407A
Other languages
English (en)
Korean (ko)
Other versions
KR20210049131A (ko
Inventor
빈 리
Original Assignee
우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니
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 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 filed Critical 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니
Publication of KR20210049131A publication Critical patent/KR20210049131A/ko
Application granted granted Critical
Publication of KR102612148B1 publication Critical patent/KR102612148B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • C12N15/52Genes encoding for enzymes or proenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y306/00Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03005Zn2+-exporting ATPase (3.6.3.5)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10Cells modified by introduction of foreign genetic material
    • 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)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y306/00Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/05Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
    • C12Y306/05005Dynamin GTPase (3.6.5.5)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y306/00Hydrolases acting on acid anhydrides (3.6)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
KR1020217008407A 2018-08-23 2019-08-23 재조합 인간 II형 미토콘드리아 다이네인-유사 GTPase의 유전자 서열 및 이의 용도 Active KR102612148B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN201810968173.0A CN110857440B (zh) 2018-08-23 2018-08-23 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
CN201810968173.0 2018-08-23
PCT/CN2019/102352 WO2020038473A1 (zh) 2018-08-23 2019-08-23 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用

Publications (2)

Publication Number Publication Date
KR20210049131A KR20210049131A (ko) 2021-05-04
KR102612148B1 true KR102612148B1 (ko) 2023-12-12

Family

ID=69592310

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217008407A Active KR102612148B1 (ko) 2018-08-23 2019-08-23 재조합 인간 II형 미토콘드리아 다이네인-유사 GTPase의 유전자 서열 및 이의 용도

Country Status (11)

Country Link
US (1) US11459584B2 (https=)
EP (1) EP3851535A4 (https=)
JP (1) JP7285022B2 (https=)
KR (1) KR102612148B1 (https=)
CN (1) CN110857440B (https=)
AU (1) AU2019323501B2 (https=)
BR (1) BR112021002566A2 (https=)
CA (1) CA3110153A1 (https=)
MX (1) MX2021002135A (https=)
SG (1) SG11202101236WA (https=)
WO (1) WO2020038473A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110857440B (zh) 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
CN111849998A (zh) * 2020-07-29 2020-10-30 武汉纽福斯生物科技有限公司 编码人卵黄状黄斑病蛋白1的核酸分子及其应用
CN117904156A (zh) * 2022-10-17 2024-04-19 武汉纽福斯生物科技有限公司 人线粒体动力蛋白样gtp酶及应用
WO2024226729A2 (en) * 2023-04-26 2024-10-31 Astellas Gene Therapies, Inc. Compositions and methods for opa1 gene therapy
CN117304293B (zh) * 2023-10-07 2025-08-08 浙江大学 一种鸡opa1抗原决定簇及其应用

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
EP1771571A2 (en) 2004-07-30 2007-04-11 Targeted Genetics Corporation Recombinant aav based vaccine methods
PT1880008E (pt) * 2005-05-03 2015-08-27 Inserm Inst Nat De La Santé Et De La Rech Médicale Expressão de proteína mitocondrial através de uma abordagem alotópica melhorada
WO2007134818A2 (en) * 2006-05-18 2007-11-29 Univ Muenchen L Maximilians Method for diagnosing mitochondrial dysfunction
CN102634527B (zh) * 2012-04-11 2013-11-06 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
AU2013293270B2 (en) * 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
AR095983A1 (es) * 2013-04-03 2015-11-25 Aliophtha Ag Factores artificiales de transcripción para el tratamiento de enfermedades causadas por haploinsuficiencia de opa1
CN104450747B (zh) * 2014-09-23 2018-02-09 武汉纽福斯生物科技有限公司 用于治疗Leber遗传性视神经病变的重组腺相关病毒‑NADH脱氢酶亚单位4基因全长以及药剂
EP3289080B1 (en) * 2015-04-30 2021-08-25 The Trustees of Columbia University in the City of New York Gene therapy for autosomal dominant diseases
WO2017191274A2 (en) 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
EP3548039B1 (en) * 2016-12-04 2023-07-19 Alavi Khorassani Moghadam, Marcel Victor Ribavirin for use in the treatment of a mitochondrial disease
WO2018146588A1 (en) 2017-02-08 2018-08-16 Friedrich Miescher Institute For Biomedical Research Synp88, a promoter for the specific expression of genes in retinal ganglion cells
CN111068071A (zh) * 2018-10-22 2020-04-28 武汉纽福斯生物科技有限公司 基因治疗Leber遗传学视神经病变
CN110857440B (zh) 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Emmanuelle Sarzi 등, Sci. Rep., 제8권, 논문번호 2468, 내부페이지 1-6 (2018)
Pasqualina Colella 등, Mol. Ther. Methods Clin. Dev., 제8권, 페이지 87-104 (2017)

Also Published As

Publication number Publication date
CN110857440B (zh) 2021-02-19
EP3851535A1 (en) 2021-07-21
SG11202101236WA (en) 2021-03-30
BR112021002566A2 (pt) 2021-06-22
JP2021533801A (ja) 2021-12-09
CA3110153A1 (en) 2020-02-27
AU2019323501B2 (en) 2023-08-03
US11459584B2 (en) 2022-10-04
CN110857440A (zh) 2020-03-03
AU2019323501A1 (en) 2021-02-25
EP3851535A4 (en) 2022-12-14
MX2021002135A (es) 2021-06-23
WO2020038473A1 (zh) 2020-02-27
US20210180086A1 (en) 2021-06-17
KR20210049131A (ko) 2021-05-04
JP7285022B2 (ja) 2023-06-01

Similar Documents

Publication Publication Date Title
KR102612148B1 (ko) 재조합 인간 II형 미토콘드리아 다이네인-유사 GTPase의 유전자 서열 및 이의 용도
JP2024015194A (ja) アデノ随伴ウイルス変異キャプシドおよび血管新生の阻害のための使用
KR102537394B1 (ko) 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
CN111621502B (zh) 视网膜劈裂蛋白的编码序列、其表达载体构建及其应用
KR20190005223A (ko) 아데노-관련 바이러스 변이체 캡시드 및 그 용도
JP7618287B2 (ja) 医薬の製造におけるCYP4V2およびRdCVFの使用
US20210347834A1 (en) Gene therapy vector for treating retinitis pigmentosa disease
CN114127296B (zh) Ube3a基因和表达盒及其应用
CN113025633A (zh) 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
KR20230061372A (ko) 코돈 최적화된 rpgrorf15 유전자 및 이의 용도
CN110699367B (zh) 编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
CN111926021B (zh) 重组人norrin胱氨酸结生长因子表达载体及其应用
WO2020077756A1 (zh) Nd4蛋白的编码序列及其应用
WO2020010491A1 (zh) 编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
WO2020000641A1 (zh) 编码人nadh脱氢酶亚单位蛋白的核酸及其应用
CN111518813B (zh) 视紫红质的编码序列、其表达载体构建及其应用
EP4163375A1 (en) Expression vector of human nuclear factor e2-related factor 2 and application of expression vector
HK40056038A (en) Recombinant human type ii mitochondrial dynein-like gtpase gene sequence and application thereof
CN111909935B (zh) 重组人卷曲蛋白受体4(fzd4)的表达载体及其应用
WO2024083013A1 (zh) 人线粒体动力蛋白样gtp酶及应用
HK40086320A (en) Expression vector of human nuclear factor e2-related factor 2 and application of expression vector

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20210322

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20220819

Comment text: Request for Examination of Application

PA0302 Request for accelerated examination

Patent event date: 20220819

Patent event code: PA03022R01D

Comment text: Request for Accelerated Examination

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20221223

Patent event code: PE09021S01D

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20230918

GRNT Written decision to grant
PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20231206

Patent event code: PR07011E01D

PR1002 Payment of registration fee

Payment date: 20231207

End annual number: 3

Start annual number: 1

PG1601 Publication of registration