JP7420558B2 - 自己不活性化ウイルスベクター - Google Patents

自己不活性化ウイルスベクター Download PDF

Info

Publication number
JP7420558B2
JP7420558B2 JP2019560380A JP2019560380A JP7420558B2 JP 7420558 B2 JP7420558 B2 JP 7420558B2 JP 2019560380 A JP2019560380 A JP 2019560380A JP 2019560380 A JP2019560380 A JP 2019560380A JP 7420558 B2 JP7420558 B2 JP 7420558B2
Authority
JP
Japan
Prior art keywords
protein
vector genome
vector
viral
cognate
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
JP2019560380A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020518265A5 (https=
JP2020518265A (ja
Inventor
トリポディ,マルコ
チャバッティ,エルネスト
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.)
United Kingdom Research and Innovation
Original Assignee
United Kingdom Research and Innovation
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 United Kingdom Research and Innovation filed Critical United Kingdom Research and Innovation
Publication of JP2020518265A publication Critical patent/JP2020518265A/ja
Publication of JP2020518265A5 publication Critical patent/JP2020518265A5/ja
Application granted granted Critical
Publication of JP7420558B2 publication Critical patent/JP7420558B2/ja
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
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/08RNA viruses
    • C07K14/145Rhabdoviridae, e.g. rabies virus, Duvenhage virus, Mokola virus or vesicular stomatitis virus
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/20Animal model comprising regulated expression system
    • A01K2217/206Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0393Animal model comprising a reporter system for screening tests
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/50Fusion polypeptide containing protease site
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/95Fusion polypeptide containing a motif/fusion for degradation (ubiquitin fusions, PEST sequence)
    • 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
    • C12N2760/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011Details
    • C12N2760/20011Rhabdoviridae
    • C12N2760/20111Lyssavirus, e.g. rabies virus
    • C12N2760/20141Use of virus, viral particle or viral elements as a vector
    • C12N2760/20143Use 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
    • C12N2770/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011Details
    • C12N2770/00022New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/609Vectors comprising as targeting moiety peptide derived from defined protein from viruses positive strand RNA viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Virology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (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)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2019560380A 2017-05-02 2018-05-02 自己不活性化ウイルスベクター Active JP7420558B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1706945.1A GB201706945D0 (en) 2017-05-02 2017-05-02 Self-inactivating viral vector
GB1706945.1 2017-05-02
PCT/GB2018/051166 WO2018203049A1 (en) 2017-05-02 2018-05-02 Self-inactivating viral vector

Publications (3)

Publication Number Publication Date
JP2020518265A JP2020518265A (ja) 2020-06-25
JP2020518265A5 JP2020518265A5 (https=) 2021-07-26
JP7420558B2 true JP7420558B2 (ja) 2024-01-23

Family

ID=59011153

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019560380A Active JP7420558B2 (ja) 2017-05-02 2018-05-02 自己不活性化ウイルスベクター

Country Status (10)

Country Link
US (1) US12203092B2 (https=)
EP (1) EP3619314B1 (https=)
JP (1) JP7420558B2 (https=)
KR (1) KR20200015900A (https=)
CN (1) CN110914439B (https=)
AU (1) AU2018262281B2 (https=)
BR (1) BR112019023003A2 (https=)
CA (1) CA3060675A1 (https=)
GB (1) GB201706945D0 (https=)
WO (1) WO2018203049A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021004389A1 (zh) * 2019-07-05 2021-01-14 司龙龙 蛋白水解靶向病毒、其活疫苗及其制备方法和用途
CA3208612A1 (en) * 2021-02-19 2022-08-25 Beam Therapeutics Inc. Recombinant rabies viruses for gene therapy
AU2022264328A1 (en) * 2021-04-27 2023-11-16 The Henry M. Jackson Foundation For The Advancement Of Military Medicine, Inc. Recombinant cedar virus chimeras
CN115704012A (zh) * 2021-08-12 2023-02-17 中国科学院深圳先进技术研究院 一种含有降解决定子的重组病毒及其制备方法和应用
CA3230629A1 (en) * 2021-09-08 2023-03-16 Beam Therapeutics Inc. Viral guide rna delivery
CN116262911B (zh) * 2021-12-14 2026-02-17 中国科学院深圳先进技术研究院 一种减毒、复制可控的顺向hsv示踪系统及其构建方法和应用
CN114058641B (zh) * 2022-01-17 2022-04-01 苏州大学 一种载体系统和应用及通过其降解目的蛋白的方法
CN116676273A (zh) * 2022-02-22 2023-09-01 中国科学院深圳先进技术研究院 一种蛋白水解靶向流感病毒及其制备方法和应用
CN120366391A (zh) * 2023-04-18 2025-07-25 上海本导基因技术有限公司 一种用于治疗亨廷顿疾病的慢病毒样颗粒
CN117867029B (zh) * 2023-12-29 2024-12-20 复百澳(苏州)生物医药科技有限公司 一种狂犬亚型假病毒的制备方法及应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016125364A1 (ja) 2015-02-02 2016-08-11 株式会社Idファーマ 改良されたマイナス鎖rnaウイルスベクター
WO2017004022A2 (en) 2015-06-29 2017-01-05 The Board Of Trustees Of The Leland Stanford Junior University Degron fusion constructs and methods for controlling protein production

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUP0500722A2 (hu) * 2001-07-20 2005-11-28 The University of Georgia Research Foundation, Inc@Á Attenuált veszettség vírus, a veszettség elleni vakcináláshoz és a központi idegrendszer génterápiájához egy nukleoprotein mutációval a foszforilezés helyén
US7608682B2 (en) 2002-10-30 2009-10-27 Meso Scale Technologies Llc Substrates of N-end rule ubiquitylation and methods for measuring the ubiquitylation of these substrates
US9212359B2 (en) 2011-06-23 2015-12-15 Sanford Jay Silverman Systems for induction of gene expression and protein depletion in yeast

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016125364A1 (ja) 2015-02-02 2016-08-11 株式会社Idファーマ 改良されたマイナス鎖rnaウイルスベクター
WO2017004022A2 (en) 2015-06-29 2017-01-05 The Board Of Trustees Of The Leland Stanford Junior University Degron fusion constructs and methods for controlling protein production

Also Published As

Publication number Publication date
US12203092B2 (en) 2025-01-21
CN110914439A (zh) 2020-03-24
KR20200015900A (ko) 2020-02-13
CA3060675A1 (en) 2018-11-08
EP3619314A1 (en) 2020-03-11
WO2018203049A1 (en) 2018-11-08
GB201706945D0 (en) 2017-06-14
EP3619314B1 (en) 2021-11-10
CN110914439B (zh) 2024-05-17
AU2018262281A1 (en) 2019-11-14
AU2018262281B2 (en) 2024-05-09
JP2020518265A (ja) 2020-06-25
US20210115472A1 (en) 2021-04-22
BR112019023003A2 (pt) 2020-05-19

Similar Documents

Publication Publication Date Title
JP7420558B2 (ja) 自己不活性化ウイルスベクター
US12102691B2 (en) Methods for efficient delivery of therapeutic molecules in vitro and in vivo
Nassi et al. Neuroanatomy goes viral!
JP7709771B2 (ja) ウイルス様粒子及びその使用
CN107530399B (zh) 治疗分子在体外和体内的有效递送
CA2775151C (fr) Nouveaux polynucleotides et polypeptides chimeriques permettant la secretion d'un polypeptide d'interet en association avec des exosomes et leurs utilisations
WO2017083423A1 (en) Methods and compositions for modulating aav infection
Cannon et al. Pseudotype-dependent lentiviral transduction of astrocytes or neurons in the rat substantia nigra
JP2019516374A (ja) がんの腫瘍崩壊療法のためのvsv/ndvハイブリッドウイルス
JP7794421B2 (ja) 腫瘍を標的にする合成アデノウイルスおよびその使用
JP2024541983A (ja) Rnaプログラム可能な細胞編集のための組成物およびシステム、ならびにそれを作製および使用する方法
JP2019522034A (ja) Cas9タンパク質およびガイドRNAを含む眼疾患治療用薬学組成物
US10738282B2 (en) Use of phospholipid scramblase inhibitors for modulating inflammatory immune responses
CN118922541A (zh) 用于rna可编程细胞编辑的组合物和系统及其制备和使用方法
Stieglitz The control of neuronal activity and behavior by members of the HCN and TPC family
Levin Muscle Lim Protein in naïve and injured neurons
Groutsi Highly disabled Herpes Simplex Virus 1 vectors: Applications in CNS regeneration with emphasis on the chronically injured spinal cord

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210506

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210506

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20220222

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220328

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220608

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220928

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230130

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230501

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230725

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231025

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: 20231222

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20240111

R150 Certificate of patent or registration of utility model

Ref document number: 7420558

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150