JP5823969B2 - 心臓特異的核酸調節因子ならびにこの方法および使用 - Google Patents

心臓特異的核酸調節因子ならびにこの方法および使用 Download PDF

Info

Publication number
JP5823969B2
JP5823969B2 JP2012535849A JP2012535849A JP5823969B2 JP 5823969 B2 JP5823969 B2 JP 5823969B2 JP 2012535849 A JP2012535849 A JP 2012535849A JP 2012535849 A JP2012535849 A JP 2012535849A JP 5823969 B2 JP5823969 B2 JP 5823969B2
Authority
JP
Japan
Prior art keywords
nucleic acid
vector
heart
promoter
seq
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.)
Expired - Fee Related
Application number
JP2012535849A
Other languages
English (en)
Japanese (ja)
Other versions
JP2013509168A5 (enExample
JP2013509168A (ja
Inventor
チユア,レイ・キム
フアンデンドリースヘ,テイエリー
デ・ブレゼル,ピーテル
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.)
Life Sciences Research Partners
Original Assignee
Life Sciences Research Partners
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 Life Sciences Research Partners filed Critical Life Sciences Research Partners
Publication of JP2013509168A publication Critical patent/JP2013509168A/ja
Publication of JP2013509168A5 publication Critical patent/JP2013509168A5/ja
Application granted granted Critical
Publication of JP5823969B2 publication Critical patent/JP5823969B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • 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
    • 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
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Toxicology (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Veterinary Medicine (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2012535849A 2009-10-29 2010-10-29 心臓特異的核酸調節因子ならびにこの方法および使用 Expired - Fee Related JP5823969B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP09174519.0 2009-10-29
EP09174519 2009-10-29
PCT/EP2010/066474 WO2011051450A1 (en) 2009-10-29 2010-10-29 Cardiac-specific nucleic acid regulatory elements and methods and use thereof

Publications (3)

Publication Number Publication Date
JP2013509168A JP2013509168A (ja) 2013-03-14
JP2013509168A5 JP2013509168A5 (enExample) 2013-12-19
JP5823969B2 true JP5823969B2 (ja) 2015-11-25

Family

ID=43414855

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2012535849A Expired - Fee Related JP5823969B2 (ja) 2009-10-29 2010-10-29 心臓特異的核酸調節因子ならびにこの方法および使用

Country Status (6)

Country Link
US (1) US9353164B2 (enExample)
EP (1) EP2494058B1 (enExample)
JP (1) JP5823969B2 (enExample)
AU (1) AU2010311376B2 (enExample)
CA (1) CA2778945A1 (enExample)
WO (1) WO2011051450A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2494058B1 (en) 2009-10-29 2017-04-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
US8859517B2 (en) 2011-08-08 2014-10-14 Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (CPVT)
US20130136729A1 (en) * 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors
US20150322439A1 (en) * 2012-11-19 2015-11-12 Nature Technology Corporation Replicative minicircle vectors with improved expression
EP3572513B1 (en) 2013-12-09 2025-09-24 Baylor College of Medicine Hippo and dystrophin complex signaling in cardiomyocyte renewal
AU2015208247B2 (en) 2014-01-21 2021-05-27 Universiteit Gent Muscle-specific nucleic acid regulatory elements and methods and use thereof
US10570183B2 (en) * 2016-04-19 2020-02-25 The Board Of Regents Of The University Of Texas System Use of a small native peptide activator of SERCA pump for treatment of heart failure and other disorders characterized by cytosolic calcium overload
WO2018178067A1 (en) 2017-03-27 2018-10-04 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
US20210343368A1 (en) * 2018-09-05 2021-11-04 Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft Method for Engineering Synthetic Cis-Regulatory DNA
JP7529286B2 (ja) 2018-10-26 2024-08-06 フリーイェ・ユニヴェルシテイト・ブリュッセル 肝特異的核酸調節エレメント並びにその方法及び使用
US20230047424A1 (en) * 2019-12-09 2023-02-16 Ucl Business Ltd Gene therapy composition and treatment for myh7-linked cardiomyopathy
EP4081643A1 (en) * 2019-12-24 2022-11-02 Asklepios Biopharmaceutical, Inc. Regulatory nucleic acid sequences
WO2021142267A1 (en) 2020-01-08 2021-07-15 Scarpmap Llc Methods and computing system for processing ultrasound image to determine health of subdermal tissue
EP4107259A1 (en) 2020-02-18 2022-12-28 Vrije Universiteit Brussel Novel combination of nucleic acid regulatory elements and methods and uses thereof
WO2022136388A1 (en) 2020-12-21 2022-06-30 Vrije Universiteit Brussel Cardiomyocyte-derived nucleic acid regulatory elements and methods and use thereof
TW202317768A (zh) 2021-07-08 2023-05-01 美商特納亞治療股份有限公司 用於基因療法的優化表現卡匣
AR126660A1 (es) 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2797273B1 (fr) * 1999-08-06 2004-01-02 Pasteur Institut Polynucleotides dirigeant l'activation de l'expression d'un gene dans le coeur et ses applications a la therapie genique
AU2001234470A1 (en) * 2000-01-14 2001-07-24 Beth Israel Deaconess Medical Center Cardiac-cell specific enhancer elements and uses thereof
EP1303617B1 (en) * 2000-07-14 2007-09-12 The Regents Of The University Of Michigan Mutant muscle-specific enhancers
US20050004058A1 (en) * 2000-12-07 2005-01-06 Patrick Benoit Sequences upstream of the carp gene, vectors containing them and uses thereof
US7714100B2 (en) * 2004-01-27 2010-05-11 Compugen Ltd Nucleotide and amino acid sequences, and assays and methods of use thereof for diagnosis of cardiac disease
WO2006015209A2 (en) * 2004-07-29 2006-02-09 Stem Cell Innovations, Inc. Differentiation of stem cells
US20080081354A1 (en) * 2006-10-02 2008-04-03 Cardiac Pacemakers, Inc. Devices, vectors and methods for inducible ischemia cardioprotection
WO2008054713A2 (en) * 2006-10-30 2008-05-08 Thomas Jefferson University Tissue specific gene therapy treatment
WO2008126083A2 (en) * 2007-04-11 2008-10-23 Technion Research & Development Foundation Ltd. Methods for identification and selection of human embryonic stem cell derived cells
US9617548B2 (en) 2008-04-22 2017-04-11 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
EP2494058B1 (en) 2009-10-29 2017-04-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof

Also Published As

Publication number Publication date
CA2778945A1 (en) 2011-05-05
WO2011051450A1 (en) 2011-05-05
AU2010311376B2 (en) 2014-07-03
US9353164B2 (en) 2016-05-31
EP2494058A1 (en) 2012-09-05
AU2010311376A2 (en) 2012-07-12
EP2494058B1 (en) 2017-04-05
US20120252882A1 (en) 2012-10-04
AU2010311376A1 (en) 2012-05-24
JP2013509168A (ja) 2013-03-14

Similar Documents

Publication Publication Date Title
JP2013509168A (ja) 心臓特異的核酸調節因子ならびにこの方法および使用
US12065669B2 (en) Methods and compositions for modulating a genome
US12037617B2 (en) Methods and compositions for modulating a genome
US20240252682A1 (en) Hbb-modulating compositions and methods
JP7315475B2 (ja) 高活性制御エレメント
US12270029B2 (en) CFTR-modulating compositions and methods
TW202310880A (zh) 靶向肌肉之基因療法之功效改進
CN112639108A (zh) 治疗非综合征性感觉神经性听力损失的方法
CN121653188A (zh) 靶向递送vegf-c的重组腺相关病毒载体及其在促进心内膜分化治疗冠心病中的应用
JP2022512831A (ja) 網膜疾患の遺伝子治療
US20240050591A1 (en) Cardiomyocyte-derived nucleic acid regulatory elements and methods and use thereof
CN115279184A (zh) B4galt1介导的功能的啮齿动物模型
AU2014240247B2 (en) Cardiac-specific nucleic acid regulatory elements and methods and use thereof
US20250163474A1 (en) Systems, methods, and compositions for targeted gene manipulation and uses thereof
CN114423863A (zh) 用于细胞遗传修饰的表达构建体
JP6153205B2 (ja) Va遺伝子破壊アデノウイルスベクターおよびそれを調製するための前駆体ベクター
WO2026025441A1 (zh) 一种用于显性遗传的视网膜色素变性基因编辑药物载体
WO2025129095A1 (en) Compositions and methods for allele-specific myh7 gene editing
HK40093831A (en) Rho-r135w-adrp gene editing drug based on gene editing
LLADO SANTAEULARIA THERAPEUTIC GENOME EDITING IN RETINA AND LIVER
EP4646479A2 (en) Trac and b2m modulating compositions and methods

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20131028

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20131028

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20141126

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20141222

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20150317

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150619

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: 20150915

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20151008

R150 Certificate of patent or registration of utility model

Ref document number: 5823969

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

LAPS Cancellation because of no payment of annual fees