JP2004506658A - アデノ随伴ウイルスを介した血管形成因子のデリバリー - Google Patents

アデノ随伴ウイルスを介した血管形成因子のデリバリー Download PDF

Info

Publication number
JP2004506658A
JP2004506658A JP2002519615A JP2002519615A JP2004506658A JP 2004506658 A JP2004506658 A JP 2004506658A JP 2002519615 A JP2002519615 A JP 2002519615A JP 2002519615 A JP2002519615 A JP 2002519615A JP 2004506658 A JP2004506658 A JP 2004506658A
Authority
JP
Japan
Prior art keywords
muscle
raav
vegf
therapeutic effect
aav
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
Application number
JP2002519615A
Other languages
English (en)
Japanese (ja)
Other versions
JP2004506658A5 (https=
Inventor
小澤 敬也
新保 昌久
池田 宇一
前田 喜一
島田 和幸
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.)
Individual
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of JP2004506658A publication Critical patent/JP2004506658A/ja
Publication of JP2004506658A5 publication Critical patent/JP2004506658A5/ja
Pending legal-status Critical Current

Links

Images

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
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/1825Fibroblast growth factor [FGF]
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/1858Platelet-derived growth factor [PDGF]
    • A61K38/1866Vascular endothelial growth factor [VEGF]
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/1891Angiogenesic factors; Angiogenin
    • 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
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Vascular Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Urology & Nephrology (AREA)
JP2002519615A 2000-08-17 2001-08-17 アデノ随伴ウイルスを介した血管形成因子のデリバリー Pending JP2004506658A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US22605600P 2000-08-17 2000-08-17
PCT/IB2001/001902 WO2002014487A2 (en) 2000-08-17 2001-08-17 Adeno-associated virus-mediated delivery of angiogenic factors

Publications (2)

Publication Number Publication Date
JP2004506658A true JP2004506658A (ja) 2004-03-04
JP2004506658A5 JP2004506658A5 (https=) 2008-10-09

Family

ID=22847367

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2002519615A Pending JP2004506658A (ja) 2000-08-17 2001-08-17 アデノ随伴ウイルスを介した血管形成因子のデリバリー

Country Status (9)

Country Link
US (2) US20020111324A1 (https=)
EP (1) EP1311699B1 (https=)
JP (1) JP2004506658A (https=)
AT (1) ATE366319T1 (https=)
AU (1) AU2001294096A1 (https=)
CA (1) CA2419245A1 (https=)
DE (1) DE60129229T2 (https=)
ES (1) ES2288993T3 (https=)
WO (1) WO2002014487A2 (https=)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2406687A1 (en) * 2001-11-09 2003-05-09 Transgene S.A. Chimeric promoters for controlling expression in muscle cells
JP2005528362A (ja) * 2002-03-21 2005-09-22 ユニヴァーシティ オヴ フロリダ 血管新生の調節法
US20030198620A1 (en) * 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
CN100387723C (zh) * 2005-12-02 2008-05-14 浙江大学 血管生长素基因重组腺相关病毒载体及制备方法和用途
GB0821834D0 (en) * 2008-11-28 2009-01-07 Iti Scotland Ltd Calibration and control method
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
CA2973096A1 (en) 2015-01-06 2016-07-14 Cardiovascular Biotherapeutics, Inc. Angiogenic treatment of ischemic heart disease
AU2016220304B2 (en) 2015-02-16 2019-07-25 Venturis Therapeutics, Inc. Therapeutic angiogenesis for treating erectile conditions
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. ハンチントン病治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Aavを送達するための組成物および方法
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
EP4433490A2 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1016726A1 (en) * 1998-12-30 2000-07-05 Introgene B.V. Gene therapy to promote angiogenesis
WO2000038518A1 (en) * 1998-12-28 2000-07-06 Arch Development Corporation Efficient and stable (in vivo) gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH0764893A (ja) * 1993-08-31 1995-03-10 Canon Inc ネットワーク・システム
US20030148968A1 (en) * 1995-02-28 2003-08-07 Hammond H. Kirk Techniques and compositions for treating cardiovascular disease by in vivo gene delivery
US6162796A (en) * 1995-09-27 2000-12-19 The Rockefeller University Method for transferring genes to the heart using AAV vectors
US6004797A (en) * 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
WO1997032990A1 (en) * 1996-03-04 1997-09-12 Targeted Genetics Corporation Methods for transducing cells in blood vessels using recombinant aav vectors
US6013780A (en) * 1996-09-06 2000-01-11 Technion Research & Development Co. Ltd. VEGF145 expression vectors
JP2002502608A (ja) * 1998-02-06 2002-01-29 コラテラル・セラピューティックス・インコーポレイテッド 血管新生促進因子である血管内皮細胞成長因子:vegfの変異体
JP2003501092A (ja) * 1999-06-07 2003-01-14 エドワーズ ライフサイエンシーズ コーポレイション 標的化新脈管形成
US7223406B2 (en) * 2000-07-21 2007-05-29 The Regents Of The University Of California Methods and compositions for preventing and treating male erectile dysfunction and female sexual arousal disorder

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000038518A1 (en) * 1998-12-28 2000-07-06 Arch Development Corporation Efficient and stable (in vivo) gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
EP1016726A1 (en) * 1998-12-30 2000-07-05 Introgene B.V. Gene therapy to promote angiogenesis

Also Published As

Publication number Publication date
WO2002014487A2 (en) 2002-02-21
EP1311699A2 (en) 2003-05-21
DE60129229D1 (de) 2007-08-16
EP1311699B1 (en) 2007-07-04
US20020111324A1 (en) 2002-08-15
AU2001294096A1 (en) 2002-02-25
ES2288993T3 (es) 2008-02-01
US20070128163A1 (en) 2007-06-07
CA2419245A1 (en) 2002-02-21
WO2002014487A3 (en) 2003-03-13
ATE366319T1 (de) 2007-07-15
DE60129229T2 (de) 2008-05-08

Similar Documents

Publication Publication Date Title
EP1311699B1 (en) Adeno-associated virus-mediated delivery of angiogenic factors
CN111902539B (zh) 杂合调控元件
US6162796A (en) Method for transferring genes to the heart using AAV vectors
JP6759177B2 (ja) 減少した免疫反応性を有するaavビリオン、およびその使用
US6447768B1 (en) Methods of gene therapy with a DNA sequence encoding NOS
JP3755827B2 (ja) 組み込み可能な組み換えアデノウィルス、それらの製造及びそれらの治療的利用
US10046067B2 (en) Decorin gene delivery system and cancer treatment
AU2025206429A1 (en) Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
US7655467B2 (en) Compositions and methods for systemic nucleic acid sequence delivery
US6245330B1 (en) Recombinant adenoviruses coding for glial-derived neurotrophic factor (GDNF)
WO2002089856A1 (en) Techniques and compositions for treating cardiovascular disease by (in vivo) gene delivery
JP2015503924A (ja) 遺伝子導入のための方法及び組成物
JP4520569B2 (ja) 拡張型心筋症の遺伝子治療剤
JP4863874B2 (ja) 関節リウマチのインビボ遺伝子治療のためのaavベクター
EP4410988A1 (en) An aav2-vector variant for targeted transfer of genes
JP7750581B2 (ja) アデノ随伴ウイルスカプシドタンパク質の突然変異体
KR20090035711A (ko) 유전자 치료를 위한 아데노-관련 바이러스 벡터의 전방 심외막 관상에의 연장 주입
EP1870473A1 (en) Adeno-associated virus-mediated delivery of angiogenic factors
WO2002046220A2 (en) Sequences upstream of the carp gene, vectors containing them and uses thereof
AU2002226400A1 (en) Sequences upstream of the carp gene, vectors containing them and uses thereof
US20060099183A1 (en) Use of recombinant adeno-associated virus vector (rAAV) for the prevention of smooth muscle cell proliferation in a vascular graft
EP1016727A1 (en) Gene therapy to promote angiogenesis.
EP4658800A1 (en) An aav9 capsid variant for targeted gene transfer

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20080818

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20080818

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20110426

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20111025