AU2010311376B2 - Cardiac-specific nucleic acid regulatory elements and methods and use thereof - Google Patents

Cardiac-specific nucleic acid regulatory elements and methods and use thereof Download PDF

Info

Publication number
AU2010311376B2
AU2010311376B2 AU2010311376A AU2010311376A AU2010311376B2 AU 2010311376 B2 AU2010311376 B2 AU 2010311376B2 AU 2010311376 A AU2010311376 A AU 2010311376A AU 2010311376 A AU2010311376 A AU 2010311376A AU 2010311376 B2 AU2010311376 B2 AU 2010311376B2
Authority
AU
Australia
Prior art keywords
nucleic acid
seq
vector
promoter
expression
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.)
Ceased
Application number
AU2010311376A
Other languages
English (en)
Other versions
AU2010311376A1 (en
AU2010311376A2 (en
Inventor
Lay Khim Chuah
Pieter De Bleser
Thierry Vandendriessche
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.)
Universiteit Gent
Vlaams Instituut voor Biotechnologie VIB
Life Sciences Research Partners vzw
Original Assignee
Universiteit Gent
Vlaams Instituut voor Biotechnologie VIB
Life Sciences Research Partners vzw
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 Universiteit Gent, Vlaams Instituut voor Biotechnologie VIB, Life Sciences Research Partners vzw filed Critical Universiteit Gent
Publication of AU2010311376A1 publication Critical patent/AU2010311376A1/en
Publication of AU2010311376A2 publication Critical patent/AU2010311376A2/en
Application granted granted Critical
Publication of AU2010311376B2 publication Critical patent/AU2010311376B2/en
Priority to AU2014240247A priority Critical patent/AU2014240247B2/en
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Cardiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (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)
AU2010311376A 2009-10-29 2010-10-29 Cardiac-specific nucleic acid regulatory elements and methods and use thereof Ceased AU2010311376B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2014240247A AU2014240247B2 (en) 2009-10-29 2014-10-02 Cardiac-specific nucleic acid regulatory elements and methods and use thereof

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP09174519 2009-10-29
EP09174519.0 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2014240247A Division AU2014240247B2 (en) 2009-10-29 2014-10-02 Cardiac-specific nucleic acid regulatory elements and methods and use thereof

Publications (3)

Publication Number Publication Date
AU2010311376A1 AU2010311376A1 (en) 2012-05-24
AU2010311376A2 AU2010311376A2 (en) 2012-07-12
AU2010311376B2 true AU2010311376B2 (en) 2014-07-03

Family

ID=43414855

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2010311376A Ceased AU2010311376B2 (en) 2009-10-29 2010-10-29 Cardiac-specific nucleic acid regulatory elements and methods and use thereof

Country Status (6)

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

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011051450A1 (en) 2009-10-29 2011-05-05 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
WO2014077863A1 (en) * 2012-11-19 2014-05-22 Nature Technology Corporation Replicative minicircle vectors with improved expression
JP6707028B2 (ja) 2013-12-09 2020-06-10 ベイラー カレッジ オブ メディスンＢａｙｌｏｒ Ｃｏｌｌｅｇｅ Ｏｆ Ｍｅｄｉｃｉｎｅ 心筋細胞新生におけるｈｉｐｐｏ及びジストロフィン複合体シグナル伝達
PL3097197T3 (pl) 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
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
EP4073257A1 (en) * 2019-12-09 2022-10-19 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
EP4087489B1 (en) 2020-01-08 2025-05-14 Vitruvia Holdings Inc. Methods and computing system for processing ultrasound image to determine health of subdermal tissue
JP7744027B2 (ja) 2020-02-18 2025-09-25 フレイエ ユニヴェルシテイト ブリュッセル 核酸調節エレメントの新規な組合せ並びにその方法及び使用
JP2024502257A (ja) * 2020-12-21 2024-01-18 フレイエ ユニヴェルシテイト ブリュッセル 心筋細胞由来核酸調節エレメント並びにその方法及び使用
KR20240032971A (ko) 2021-07-08 2024-03-12 테나야 테라퓨틱스, 인코포레이티드 유전자 요법을 위한 최적화된 발현 카세트
AR126660A1 (es) 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001011064A2 (fr) * 1999-08-06 2001-02-15 Institut Pasteur Polynucleotides dirigeant l'activation de l'expression d'un gene dans le coeur et ses applications a la therapie genique
WO2001051006A2 (en) * 2000-01-14 2001-07-19 Beth Israel Deaconess Medical Center Cardiac-cell specific enhancer elements and uses thereof
US20050004058A1 (en) * 2000-12-07 2005-01-06 Patrick Benoit Sequences upstream of the carp gene, vectors containing them and uses thereof
WO2006015209A2 (en) * 2004-07-29 2006-02-09 Stem Cell Innovations, Inc. Differentiation of stem cells
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

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE373095T1 (de) * 2000-07-14 2007-09-15 Univ Michigan Mutierte muskelspezifische enhancer
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
US20080081354A1 (en) * 2006-10-02 2008-04-03 Cardiac Pacemakers, Inc. Devices, vectors and methods for inducible ischemia cardioprotection
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
WO2011051450A1 (en) 2009-10-29 2011-05-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001011064A2 (fr) * 1999-08-06 2001-02-15 Institut Pasteur Polynucleotides dirigeant l'activation de l'expression d'un gene dans le coeur et ses applications a la therapie genique
WO2001051006A2 (en) * 2000-01-14 2001-07-19 Beth Israel Deaconess Medical Center Cardiac-cell specific enhancer elements and uses thereof
US20050004058A1 (en) * 2000-12-07 2005-01-06 Patrick Benoit Sequences upstream of the carp gene, vectors containing them and uses thereof
WO2006015209A2 (en) * 2004-07-29 2006-02-09 Stem Cell Innovations, Inc. Differentiation of stem cells
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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
EMBL Accession No. M24242 14 November 2006 *
Kurabayashi, M. et al., Journal of Biological Chemistry. 1990 vol. 265 no. 31 pages 19271-19278 *

Also Published As

Publication number Publication date
EP2494058B1 (en) 2017-04-05
JP2013509168A (ja) 2013-03-14
JP5823969B2 (ja) 2015-11-25
EP2494058A1 (en) 2012-09-05
CA2778945A1 (en) 2011-05-05
US9353164B2 (en) 2016-05-31
AU2010311376A1 (en) 2012-05-24
AU2010311376A2 (en) 2012-07-12
WO2011051450A1 (en) 2011-05-05
US20120252882A1 (en) 2012-10-04

Similar Documents

Publication Publication Date Title
AU2010311376B2 (en) Cardiac-specific nucleic acid regulatory elements and methods and use thereof
US12409236B2 (en) Liver-specific nucleic acid regulatory elements and methods and use thereof
JP2023093487A (ja) 横隔膜特異的核酸調節エレメントならびにその方法および使用
CN112313334A (zh) 用以编辑血红蛋白相关突变的同源定向修复模板设计和递送
WO2023006890A1 (en) Improving the efficacy of muscle-targeted gene therapy
TW202421787A (zh) 保護心臟之心臟病療法
EP4368209A1 (en) Cardiac vascular endothelial cell-derived nucleic acid regulatory elements and methods and use thereof
TW202129002A (zh) 用於myh7關聯之心肌病之基因療法組合物及治療
AU2014240247B2 (en) Cardiac-specific nucleic acid regulatory elements and methods and use thereof
US20240050591A1 (en) Cardiomyocyte-derived nucleic acid regulatory elements and methods and use thereof
US20260027236A1 (en) Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2025129095A1 (en) Compositions and methods for allele-specific myh7 gene editing
WO2026080685A1 (en) Guide rnas, vectors, and virions for targeting mutations in the pln gene
HK40090887A (zh) 治疗心贜疾患和充血性心力衰竭以及施用aav载体的方法
HK40014510A (en) Liver-specific nucleic acid regulatory elements and methods and use thereof
HK40046220A (en) Homology-directed repair template design and delivery to edit hemoglobin-related mutations

Legal Events

Date Code Title Description
DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS AS SHOWN IN THE STATEMENT(S) FILED 25 JUN 2012

FGA Letters patent sealed or granted (standard patent)
MK14 Patent ceased section 143(a) (annual fees not paid) or expired