PT3684422T - Terapia génica de conexina-43 para restabelecer a função elétrica e cardíaca, e a estrutura cardíaca, na cardiomiopatia arritmogénica ventricular direita - Google Patents

Terapia génica de conexina-43 para restabelecer a função elétrica e cardíaca, e a estrutura cardíaca, na cardiomiopatia arritmogénica ventricular direita

Info

Publication number
PT3684422T
PT3684422T PT188586200T PT18858620T PT3684422T PT 3684422 T PT3684422 T PT 3684422T PT 188586200 T PT188586200 T PT 188586200T PT 18858620 T PT18858620 T PT 18858620T PT 3684422 T PT3684422 T PT 3684422T
Authority
PT
Portugal
Prior art keywords
cardiac
gene therapy
right ventricular
arrhythmogenic right
therapy strategy
Prior art date
Application number
PT188586200T
Other languages
English (en)
Original Assignee
Univ California
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 Univ California filed Critical Univ California
Publication of PT3684422T publication Critical patent/PT3684422T/pt

Links

Classifications

    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • 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
    • A61P9/06—Antiarrhythmics
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0375—Animal model for cardiovascular diseases
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PT188586200T 2017-09-20 2018-09-20 Terapia génica de conexina-43 para restabelecer a função elétrica e cardíaca, e a estrutura cardíaca, na cardiomiopatia arritmogénica ventricular direita PT3684422T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762560989P 2017-09-20 2017-09-20

Publications (1)

Publication Number Publication Date
PT3684422T true PT3684422T (pt) 2024-03-27

Family

ID=65810964

Family Applications (1)

Application Number Title Priority Date Filing Date
PT188586200T PT3684422T (pt) 2017-09-20 2018-09-20 Terapia génica de conexina-43 para restabelecer a função elétrica e cardíaca, e a estrutura cardíaca, na cardiomiopatia arritmogénica ventricular direita

Country Status (11)

Country Link
US (1) US20200215155A1 (pt)
EP (2) EP3684422B1 (pt)
JP (2) JP7418826B2 (pt)
CN (1) CN111107880A (pt)
AU (1) AU2018335401A1 (pt)
CA (1) CA3076227A1 (pt)
DK (1) DK3684422T5 (pt)
ES (1) ES2976559T3 (pt)
IL (1) IL273448A (pt)
PT (1) PT3684422T (pt)
WO (1) WO2019060619A1 (pt)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7220717B2 (ja) 2017-12-22 2023-02-10 マジック リープ， インコーポレイテッド レイキャスティングおよびライブ深度を使用したオクルージョンレンダリングの方法
SG11202110607WA (en) 2019-04-01 2021-10-28 Tenaya Therapeutics Inc Adeno-associated virus with engineered capsid
JP2022549140A (ja) * 2019-09-20 2022-11-24 ユーシーエル ビジネス リミテッド 遺伝子療法組成物および不整脈源性右心室心筋症の治療
WO2021187380A1 (ja) * 2020-03-16 2021-09-23 国立大学法人大阪大学 不整脈源性心筋症患者由来の多能性幹細胞およびその利用ならびに不整脈源性心筋症治療用医薬
EP4192487A4 (en) 2020-08-07 2024-10-02 Spacecraft Seven Llc GENE THERAPY FOR PLAKOPHILIN-2 (PKP2) INVOLVING AAV VECTOR
CN115698304A (zh) * 2020-10-09 2023-02-03 特纳亚治疗股份有限公司 亲斑蛋白-2基因疗法的方法和组合物
JP2024512483A (ja) * 2021-03-19 2024-03-19 ユーシーエル ビジネス リミテッド 遺伝子治療組成物及び不整脈原性右室心筋症の治療
WO2023159190A1 (en) * 2022-02-18 2023-08-24 Ginkgo Bioworks, Inc. Gene therapy for arrhythmogenic cardiomyopathy
WO2023219655A1 (en) * 2022-05-13 2023-11-16 University Of Utah Research Foundation Gja1-20k to limited cardiac arrhythmias
WO2024050464A2 (en) * 2022-08-31 2024-03-07 University Of Utah Research Foundation Gja1-20k compositions and methods for mitigating ischemia-reperfusion injury

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2324540T3 (es) 1998-05-27 2009-08-10 Genzyme Corporation Vectores aav para la fabricacion de medicamentos para la administracion potenciada por conveccion.
CN1612691A (zh) * 2001-11-08 2005-05-04 加利福尼亚大学董事会 纠正心脏传导障碍的方法和组合物
CN1703257A (zh) * 2002-11-16 2005-11-30 加利福尼亚大学董事会 心脏刺激系统和方法
WO2005085423A1 (en) * 2004-02-27 2005-09-15 Michigan State University Oct-4 and gjic expression as markers for adult human stem cells and cancer cell precursors
US7729761B2 (en) * 2004-07-14 2010-06-01 Cardiac Pacemakers, Inc. Method and apparatus for controlled gene or protein delivery
JP2008515997A (ja) * 2004-10-13 2008-05-15 メドトロニック・インコーポレーテッド 心室性頻脈を治療するための細胞的及び遺伝子的介入
JP5964538B2 (ja) * 2005-02-03 2016-08-03 コーダ セラピューティクス， インコーポレイテッド 抗コネキシン化合物およびそれらの使用
WO2009111755A2 (en) * 2008-03-07 2009-09-11 The Trustees Of Columbia University In The City Of New York Compensating for atrioventricular block using a nucleic acid encoding a sodium channel or gap junction protein
WO2009120082A2 (en) * 2008-03-27 2009-10-01 Academisch Medisch Centrum Bij De Universiteit Van Amsterdam Means and methods for influencing electrical activity of cells
EP2792742A1 (en) * 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
CN106596972B (zh) * 2016-12-16 2018-08-10 青岛科技大学 一种检测力竭运动小鼠心肌连接蛋白43含量的方法

Also Published As

Publication number Publication date
EP3684422A4 (en) 2021-06-23
US20200215155A1 (en) 2020-07-09
AU2018335401A1 (en) 2020-04-02
ES2976559T3 (es) 2024-08-05
EP3684422A1 (en) 2020-07-29
JP2020534314A (ja) 2020-11-26
JP7418826B2 (ja) 2024-01-22
JP2024050553A (ja) 2024-04-10
CA3076227A1 (en) 2019-03-28
WO2019060619A1 (en) 2019-03-28
CN111107880A (zh) 2020-05-05
EP3684422B1 (en) 2023-12-20
DK3684422T5 (en) 2024-08-05
IL273448A (en) 2020-05-31
EP4382116A1 (en) 2024-06-12
DK3684422T3 (en) 2024-03-25

Similar Documents

Publication Publication Date Title
IL273448A (en) A gene therapy strategy to restore electrical, cardiac function and heart structure in ARRHYTHMOGENIC RIGHT VENTRICULAR CARDIOMYOPATHY
IL268576A (en) Systems, catheters, and related methods for mapping, minimizing, and treating cardiac fibrillation
EP3381507C0 (en) MULTI-ELECTRODE STIMULATION THERAPY WITH REDUCED ENERGY
EP2983732A4 (en) Left ventricular cardiac assist pump and methods therefor
IL241975B (en) Cardiac synchronization performed by sensing the p-wave signal and dynamic prediction of heart action and left ventricular pacing
EP3291723C0 (en) CARDIAC IMPLANT WITH SEPTUM GRIPPER ORGAN
IL246415A0 (en) Catheter with an assembly of stacked electrode arrays
EP3188790A4 (en) Cardiac pacing
EP3119470A4 (en) Multi-electrode epicardial pacing
EP2903510A4 (en) VENTRICULAR STEERING CONTROL IN CARDIAL APPLICATIONS
IL256956B (en) Devices and methods for cardiac arrhythmia mapping
EP2833964A4 (en) MINIMALLY INVASIVE EPICARDIC CARDIAC STIMULATOR
EP3424557A4 (en) INTRAKARDIAL DEFIBRILLATION CATHETER
PL3125988T3 (pl) Zestaw elektrod, zwłaszcza do defibrylatora
EP3275471A4 (en) Three-dimensional structure for cardiac muscular tissue regeneration and manufacturing method therefor
EP2866878A4 (en) ACTIVE CARDIAC ELECTRICAL BYPASS
EP3191172A4 (en) Nerve cuff electrode for neuromodulation in large human nerve trunks
PT3074083T (pt) Dispositivo para a implantação transcutânea de elétrodos de marca-passo epicárdicos
GB201806580D0 (en) Methods and systems useful in mapping heart rhythm abnormalities
GB201601034D0 (en) Conductive paste,electrode and solar cell
EP3481323A4 (en) DEVICES FOR TREATING CARDIAC ARRHYTHMIA AND THEIR ADMINISTRATION
PL2808053T3 (pl) Biokompatybilna elektroda wszczepialna
EP3160580B8 (de) Kabelloser herzschrittmacher mit cermet-elektrode und herstellungsverfahren dafür
EP3219355A4 (en) Mesh electrode for cardiac resynchronization therapy, and manufacturing method therefor
EP3475961A4 (en) WET ELECTROLYTE CAPACITOR FOR USE IN A SUBCUTANEOUS IMPLANTABLE CARDIOVERTER DEFIBRILLATOR