IT201800004253A1 - Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria. - Google Patents

Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria. Download PDF

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Publication number
IT201800004253A1
IT201800004253A1 IT102018000004253A IT201800004253A IT201800004253A1 IT 201800004253 A1 IT201800004253 A1 IT 201800004253A1 IT 102018000004253 A IT102018000004253 A IT 102018000004253A IT 201800004253 A IT201800004253 A IT 201800004253A IT 201800004253 A1 IT201800004253 A1 IT 201800004253A1
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Italy
Prior art keywords
casq2
patient
promoter
mutation
amino acid
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IT102018000004253A
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English (en)
Italian (it)
Inventor
Silvia G Priori
Andrea Mazzanti
Marco Denegri
Carlo Napolitano
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Priority to IT102018000004253A priority Critical patent/IT201800004253A1/it
Priority to AU2019249374A priority patent/AU2019249374B2/en
Priority to ES19724606T priority patent/ES2988961T3/es
Priority to FIEP19724606.9T priority patent/FI3773744T3/fi
Priority to PT197246069T priority patent/PT3773744T/pt
Priority to JP2020552905A priority patent/JP7500429B2/ja
Priority to DK19724606.9T priority patent/DK3773744T3/da
Priority to PCT/IB2019/052817 priority patent/WO2019193563A1/en
Priority to US17/045,289 priority patent/US20210030894A1/en
Priority to IL315337A priority patent/IL315337A/en
Priority to PL19724606.9T priority patent/PL3773744T3/pl
Priority to CA3094141A priority patent/CA3094141A1/en
Priority to IL277635A priority patent/IL277635B2/en
Priority to EP24160383.6A priority patent/EP4356727A3/en
Priority to EP19724606.9A priority patent/EP3773744B1/en
Publication of IT201800004253A1 publication Critical patent/IT201800004253A1/it
Priority to JP2024091467A priority patent/JP2024119900A/ja
Priority to AU2026202418A priority patent/AU2026202418A1/en

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    • 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
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1738—Calcium binding proteins, e.g. calmodulin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • C07K14/46—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
    • C07K14/4728—Calcium binding proteins, e.g. calmodulin
    • 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
    • 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
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2267/0306—Animal model for genetic 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Animal Husbandry (AREA)
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  • Marine Sciences & Fisheries (AREA)
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IT102018000004253A 2018-04-05 2018-04-05 Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria. IT201800004253A1 (it)

Priority Applications (17)

Application Number Priority Date Filing Date Title
IT102018000004253A IT201800004253A1 (it) 2018-04-05 2018-04-05 Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria.
PCT/IB2019/052817 WO2019193563A1 (en) 2018-04-05 2019-04-05 Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia
PL19724606.9T PL3773744T3 (pl) 2018-04-05 2019-04-05 Kompozycje i sposoby leczenia wielokształtnego katecholaminergicznego częstoskurczu komorowego dziedziczonego w sposób dominujący
FIEP19724606.9T FI3773744T3 (fi) 2018-04-05 2019-04-05 Koostumukset ja menetelmät vallitsevasti perityn katekoliamiiniherkän polymorfisen kammiotakykardian hoitoon
PT197246069T PT3773744T (pt) 2018-04-05 2019-04-05 Composições e métodos para o tratamento de taquicardia ventricular polimórfica catecolaminérgica de hereditariedade dominante
JP2020552905A JP7500429B2 (ja) 2018-04-05 2019-04-05 優性遺伝性カテコラミン誘発多形性心室頻拍の治療のための組成物および方法
DK19724606.9T DK3773744T3 (da) 2018-04-05 2019-04-05 Sammensætninger og fremgangsmåder til behandling af dominant arvet katekolaminerg polymorf ventrikulær takykardi
AU2019249374A AU2019249374B2 (en) 2018-04-05 2019-04-05 Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia
US17/045,289 US20210030894A1 (en) 2018-04-05 2019-04-05 Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia
IL315337A IL315337A (en) 2018-04-05 2019-04-05 Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia
ES19724606T ES2988961T3 (es) 2018-04-05 2019-04-05 Composiciones y métodos para el tratamiento de la taquicardia ventricular polimórfica catecolaminérgica de herencia dominante
CA3094141A CA3094141A1 (en) 2018-04-05 2019-04-05 Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia
IL277635A IL277635B2 (en) 2018-04-05 2019-04-05 Compositions and methods for treating dominantly inherited catecholaminergic polymorphic ventricular tachycardia
EP24160383.6A EP4356727A3 (en) 2018-04-05 2019-04-05 Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia
EP19724606.9A EP3773744B1 (en) 2018-04-05 2019-04-05 Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia
JP2024091467A JP2024119900A (ja) 2018-04-05 2024-06-05 優性遺伝性カテコラミン誘発多形性心室頻拍の治療のための組成物および方法
AU2026202418A AU2026202418A1 (en) 2018-04-05 2026-03-31 Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
IT102018000004253A IT201800004253A1 (it) 2018-04-05 2018-04-05 Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria.

Publications (1)

Publication Number Publication Date
IT201800004253A1 true IT201800004253A1 (it) 2019-10-05

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IT102018000004253A IT201800004253A1 (it) 2018-04-05 2018-04-05 Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria.

Country Status (13)

Country Link
US (1) US20210030894A1 (https=)
EP (2) EP3773744B1 (https=)
JP (2) JP7500429B2 (https=)
AU (2) AU2019249374B2 (https=)
CA (1) CA3094141A1 (https=)
DK (1) DK3773744T3 (https=)
ES (1) ES2988961T3 (https=)
FI (1) FI3773744T3 (https=)
IL (2) IL315337A (https=)
IT (1) IT201800004253A1 (https=)
PL (1) PL3773744T3 (https=)
PT (1) PT3773744T (https=)
WO (1) WO2019193563A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2023000453A (es) * 2020-07-08 2023-02-09 Childrens Medical Center Polipeptidos de mybpc3 y usos de los mismos.
CN113957075B (zh) * 2021-11-22 2023-04-07 百世诺(北京)医学检验实验室有限公司 突变的遗传性心律失常基因及其应用

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5766960A (en) 1987-07-27 1998-06-16 Australian Membrane And Biotechnology Research Institute Receptor membranes
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
MD1367C2 (ro) 1992-11-13 2000-11-30 Idec Pharmaceuticals Corporation Metode de tratament al limfomului celulelor B, anticorpi anti-CD20, hibridom.
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
US6268210B1 (en) 1998-05-27 2001-07-31 Hyseq, Inc. Sandwich arrays of biological compounds
US6232068B1 (en) 1999-01-22 2001-05-15 Rosetta Inpharmatics, Inc. Monitoring of gene expression by detecting hybridization to nucleic acid arrays using anti-heteronucleic acid antibodies
CA2407651C (en) 2000-04-27 2013-07-02 Max-Delbruck-Centrum Fur Molekulare Medizin Sleeping beauty, a transposon vector with a broad host range for the genetic transformation in vertebrates
CA2456977C (en) 2001-08-16 2011-06-07 The Trustees Of The University Of Pennsylvania Synthesis and use of reagents for improved dna lipofection and/or slow release prodrug and drug therapies
CA2891996A1 (en) 2005-10-18 2007-04-26 Duke University Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
US7741529B1 (en) 2006-05-04 2010-06-22 Priori Silvia G Transgenic animal model for catecholaminergic polymorphic ventricular tachycardia (CPVT) and use thereof
EP1995316A1 (de) 2007-05-25 2008-11-26 Qiagen GmbH Verfahren zur schonenden Zellaufreinigung, Zellgewinnung und Transfektion von Zellen
EP2009095A1 (en) 2007-06-28 2008-12-31 Innovalor AG Method of generating glucose-responsive cells
EP2215223B1 (en) 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
WO2010085699A2 (en) 2009-01-23 2010-07-29 The Johns Hopkins University Mammalian piggybac transposon and methods of use
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)
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2016083625A1 (en) * 2014-11-28 2016-06-02 Institut National De La Sante Et De La Recherche Medicale (Inserm) Nucleic acid encoding triadin for the prevention and/or treatment of heart failure
EP3417063A1 (en) * 2016-02-15 2018-12-26 Istituti Clinici Scientifici Maugeri SpA SB Method of allele specific silencing for the treatment of autosomal dominant catecholaminergic polymorphic ventricular tachycardia (cpvt)

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IL315337A (en) 2024-10-01
JP2024119900A (ja) 2024-09-03
IL277635A (en) 2020-11-30
AU2019249374A1 (en) 2020-10-22
JP2021523093A (ja) 2021-09-02
IL277635B2 (en) 2025-02-01
DK3773744T3 (da) 2024-08-26
EP4356727A3 (en) 2024-07-24
FI3773744T3 (fi) 2024-08-12
ES2988961T3 (es) 2024-11-22
AU2026202418A1 (en) 2026-04-16
EP3773744A1 (en) 2021-02-17
US20210030894A1 (en) 2021-02-04
AU2019249374B2 (en) 2026-01-08
WO2019193563A1 (en) 2019-10-10
PL3773744T3 (pl) 2024-11-04
EP3773744B1 (en) 2024-05-22
EP4356727A2 (en) 2024-04-24
CA3094141A1 (en) 2019-10-10
PT3773744T (pt) 2024-08-22
JP7500429B2 (ja) 2024-06-17
IL277635B1 (en) 2024-10-01

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