CN112384625A - 重组aav载体及其使用方法 - Google Patents
重组aav载体及其使用方法 Download PDFInfo
- Publication number
- CN112384625A CN112384625A CN201980039197.0A CN201980039197A CN112384625A CN 112384625 A CN112384625 A CN 112384625A CN 201980039197 A CN201980039197 A CN 201980039197A CN 112384625 A CN112384625 A CN 112384625A
- Authority
- CN
- China
- Prior art keywords
- seq
- sequence
- recombinant
- cox10
- recombinant aav2
- 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.)
- Pending
Links
Images
Classifications
-
- A—HUMAN NECESSITIES
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
- C12N9/0026—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5)
- C12N9/0028—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5) with NAD or NADP as acceptor (1.5.1)
-
- 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
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
- C12N9/0036—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on NADH or NADPH (1.6)
-
- 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
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0053—Oxidoreductases (1.) acting on a heme group of donors (1.9)
-
- 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
- C12N9/10—Transferases (2.)
- C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y106/00—Oxidoreductases acting on NADH or NADPH (1.6)
- C12Y106/05—Oxidoreductases acting on NADH or NADPH (1.6) with a quinone or similar compound as acceptor (1.6.5)
- C12Y106/05003—NADH dehydrogenase (ubiquinone) (1.6.5.3)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y109/00—Oxidoreductases acting on a heme group of donors (1.9)
- C12Y109/03—Oxidoreductases acting on a heme group of donors (1.9) with oxygen as acceptor (1.9.3)
- C12Y109/03001—Cytochrome-c oxidase (1.9.3.1)
-
- 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
- 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
-
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Ophthalmology & Optometry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862683501P | 2018-06-11 | 2018-06-11 | |
| US62/683,501 | 2018-06-11 | ||
| PCT/US2019/036487 WO2019241206A1 (en) | 2018-06-11 | 2019-06-11 | Recombinant aav vectors and methods of using the same |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN112384625A true CN112384625A (zh) | 2021-02-19 |
Family
ID=67138055
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980039197.0A Pending CN112384625A (zh) | 2018-06-11 | 2019-06-11 | 重组aav载体及其使用方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20210189424A1 (https=) |
| EP (1) | EP3802840A1 (https=) |
| JP (1) | JP7470057B2 (https=) |
| KR (1) | KR102946006B1 (https=) |
| CN (1) | CN112384625A (https=) |
| AU (1) | AU2019286386B2 (https=) |
| CA (1) | CA3103231A1 (https=) |
| WO (1) | WO2019241206A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20240014102A (ko) | 2018-06-29 | 2024-01-31 | 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 | 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법 |
| CA3109432A1 (en) * | 2018-08-20 | 2020-02-27 | Wuhan Neurophth Biotechnology Limited Company | Compositions and methods for treating leber's hereditary optic neuropathy |
| CN113025633B (zh) | 2019-12-09 | 2024-08-27 | 武汉纽福斯生物科技有限公司 | 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用 |
| CN115710585B (zh) * | 2021-08-23 | 2025-03-28 | 纽福斯(苏州)生物科技有限公司 | 编码nd4的核酸及其用途 |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008101233A2 (en) * | 2007-02-16 | 2008-08-21 | University Of Florida Research Foundation Inc. | Mitochondrial nucleic acid delivery systems |
| US20090306188A1 (en) * | 2005-05-03 | 2009-12-10 | Inserm (Institut National De Al Sante Et De Al Rec | Importation of mitochondrial protein by an enhanced allotopic approach |
| CN102634527A (zh) * | 2012-04-11 | 2012-08-15 | 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 |
| CN104450747A (zh) * | 2014-09-23 | 2015-03-25 | 李斌 | 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 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6218117B1 (en) * | 1998-06-15 | 2001-04-17 | Mitokor | Compositions and methods for identifying agents that quantitatively alter detectable extramitochondrial DNA:mitochondrial DNA ratios |
| DE102010013829A1 (de) * | 2010-03-26 | 2011-09-29 | Carl Zeiss Microlmaging Gmbh | Mikroskop und Verfahren zur Erfassung von Probenlicht |
| TWI775096B (zh) * | 2012-05-15 | 2022-08-21 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| KR102244434B1 (ko) * | 2014-08-11 | 2021-04-23 | 삼성전자주식회사 | 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법 |
-
2019
- 2019-06-11 CN CN201980039197.0A patent/CN112384625A/zh active Pending
- 2019-06-11 WO PCT/US2019/036487 patent/WO2019241206A1/en not_active Ceased
- 2019-06-11 US US16/973,900 patent/US20210189424A1/en active Pending
- 2019-06-11 JP JP2020569147A patent/JP7470057B2/ja active Active
- 2019-06-11 EP EP19734988.9A patent/EP3802840A1/en active Pending
- 2019-06-11 CA CA3103231A patent/CA3103231A1/en active Pending
- 2019-06-11 AU AU2019286386A patent/AU2019286386B2/en active Active
- 2019-06-11 KR KR1020217000782A patent/KR102946006B1/ko active Active
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20090306188A1 (en) * | 2005-05-03 | 2009-12-10 | Inserm (Institut National De Al Sante Et De Al Rec | Importation of mitochondrial protein by an enhanced allotopic approach |
| WO2008101233A2 (en) * | 2007-02-16 | 2008-08-21 | University Of Florida Research Foundation Inc. | Mitochondrial nucleic acid delivery systems |
| CN102634527A (zh) * | 2012-04-11 | 2012-08-15 | 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 |
| CN104450747A (zh) * | 2014-09-23 | 2015-03-25 | 李斌 | 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 |
Non-Patent Citations (3)
| Title |
|---|
| MACAULAY.V ET AL.: "Homo sapiens isolate 2_D4b2b(Tor50) mitochondrion, complete genome, ACCESSION:AY963573 REGION:10759-12136", GENBANK, vol. 1, pages 129 - 2 * |
| 刘恩岐等: "真核细胞基因表达系统", 人类疾病动物模型, pages 6 * |
| 贾战生: "肝病细胞治疗:基础与临床", vol. 1, 30 April 2005, 人民卫生出版社, pages: 409 * |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2021526834A (ja) | 2021-10-11 |
| KR102946006B1 (ko) | 2026-03-30 |
| AU2019286386B2 (en) | 2025-05-29 |
| US20210189424A1 (en) | 2021-06-24 |
| CA3103231A1 (en) | 2019-12-19 |
| AU2019286386A1 (en) | 2021-01-28 |
| WO2019241206A1 (en) | 2019-12-19 |
| AU2019286386A8 (en) | 2021-02-11 |
| EP3802840A1 (en) | 2021-04-14 |
| JP7470057B2 (ja) | 2024-04-17 |
| KR20210123285A (ko) | 2021-10-13 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| KR102946006B1 (ko) | 재조합 aav 벡터 및 그 이용 방법 | |
| KR20220022107A (ko) | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 | |
| CN108753824B (zh) | 用于治疗视网膜营养不良的病毒载体 | |
| KR102450833B1 (ko) | 혈우병 a 치료용 유전자 요법 | |
| KR102691097B1 (ko) | 표적 특이적 crispr 변이체 | |
| KR101471445B1 (ko) | 조류에서 이식유전자 발현 | |
| KR20210151916A (ko) | 뒤시엔느 근육 이영양증의 치료를 위한 aav 벡터-매개된 큰 돌연변이 핫스팟의 결실 | |
| CN110467679B (zh) | 一种融合蛋白、碱基编辑工具和方法及其应用 | |
| CN109069668B (zh) | 用于眼病的基因疗法 | |
| KR20230091894A (ko) | 부위 특이적 표적화 요소를 통한 프로그램 가능한 첨가(paste)를 사용하는 부위 특이적 유전 공학을 위한 시스템, 방법, 및 조성물 | |
| KR20220161297A (ko) | 신규 세포주 | |
| KR101449587B1 (ko) | 장기 지속형 약물 제형 | |
| CN111733174B (zh) | 一种分离的核酸分子及其用途 | |
| CN113106124B (zh) | 表达cyp4v2的aav载体及其用途 | |
| KR20220030205A (ko) | 면역 세포의 트랜스포존 기반 변형 | |
| KR20220163950A (ko) | Aav 생산을 위한 이중 이기능성 벡터 | |
| KR20210151785A (ko) | 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 | |
| KR101591887B1 (ko) | 장기 지속형 약물 제형 | |
| AU2018254529B2 (en) | Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia | |
| CN114350710A (zh) | 编码vegf拮抗剂的多核苷酸表达盒、重组病毒及相关应用 | |
| CN111500635B (zh) | 一种包含携带核酸分子的载体的试剂盒 | |
| CN112203697A (zh) | 编码氨基己糖苷酶alpha和beta亚基的双顺反子AAV载体及其用途 | |
| US5955368A (en) | Expression system for clostridium species | |
| AU780854B2 (en) | Hormone-hormone receptor complexes and nucleic acid constructs and their use in gene therapy | |
| CN101652475A (zh) | 在禽类中进行转基因表达 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PB01 | Publication | ||
| PB01 | Publication | ||
| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination |