AU2019323501B2 - Recombinant human type II mitochondrial dynein-like GTPase gene sequence and application thereof - Google Patents
Recombinant human type II mitochondrial dynein-like GTPase gene sequence and application thereof Download PDFInfo
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- AU2019323501B2 AU2019323501B2 AU2019323501A AU2019323501A AU2019323501B2 AU 2019323501 B2 AU2019323501 B2 AU 2019323501B2 AU 2019323501 A AU2019323501 A AU 2019323501A AU 2019323501 A AU2019323501 A AU 2019323501A AU 2019323501 B2 AU2019323501 B2 AU 2019323501B2
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- 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
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
- C12Y306/03005—Zn2+-exporting ATPase (3.6.3.5)
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K38/00—Medicinal preparations containing peptides
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- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal 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
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/10—Cells modified by introduction of foreign genetic material
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- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/05—Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
- C12Y306/05005—Dynamin GTPase (3.6.5.5)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
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- General Health & Medical Sciences (AREA)
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- Biotechnology (AREA)
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- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
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- 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)
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 |
|---|---|
| AU2019323501A1 AU2019323501A1 (en) | 2021-02-25 |
| AU2019323501B2 true AU2019323501B2 (en) | 2023-08-03 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2019323501A Active AU2019323501B2 (en) | 2018-08-23 | 2019-08-23 | Recombinant human type II mitochondrial dynein-like GTPase gene sequence and application thereof |
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)
| 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)
| 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 |
-
2018
- 2018-08-23 CN CN201810968173.0A patent/CN110857440B/zh active Active
-
2019
- 2019-08-23 MX MX2021002135A patent/MX2021002135A/es unknown
- 2019-08-23 AU AU2019323501A patent/AU2019323501B2/en active Active
- 2019-08-23 BR BR112021002566-9A patent/BR112021002566A2/pt not_active IP Right Cessation
- 2019-08-23 EP EP19851390.5A patent/EP3851535A4/en not_active Withdrawn
- 2019-08-23 WO PCT/CN2019/102352 patent/WO2020038473A1/zh not_active Ceased
- 2019-08-23 JP JP2021509904A patent/JP7285022B2/ja active Active
- 2019-08-23 CA CA3110153A patent/CA3110153A1/en active Pending
- 2019-08-23 SG SG11202101236WA patent/SG11202101236WA/en unknown
- 2019-08-23 KR KR1020217008407A patent/KR102612148B1/ko active Active
-
2021
- 2021-02-23 US US17/182,903 patent/US11459584B2/en active Active
Non-Patent Citations (2)
| Title |
|---|
| DATABASE Geneseq FOTIN-MLECZEK M : "human therapeutic protein OPA1 RNA SEQ ID 20917", XP055975072, retrieved from EBI * |
| DEL DOTTO VALENTINA ET AL: "Eight human OPA1 isoforms, long and short: What are they for?", BIOCHIMICA ET BIOPHYSICA ACTA. BIOENERGETICS, 1 April 2018, 1859(4):263-269, DOI: 10.1016/j.bbabio.2018.01.005 * |
Also Published As
| Publication number | Publication date |
|---|---|
| CN110857440B (zh) | 2021-02-19 |
| EP3851535A1 (en) | 2021-07-21 |
| SG11202101236WA (en) | 2021-03-30 |
| KR102612148B1 (ko) | 2023-12-12 |
| BR112021002566A2 (pt) | 2021-06-22 |
| JP2021533801A (ja) | 2021-12-09 |
| CA3110153A1 (en) | 2020-02-27 |
| 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 |
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