ES2958662T3 - Tratamiento de cardiopatía por inhibición de la acción de la proteína de anclaje a la cinasa A del músculo (mAKAP) - Google Patents

Tratamiento de cardiopatía por inhibición de la acción de la proteína de anclaje a la cinasa A del músculo (mAKAP) Download PDF

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ES2958662T3
ES2958662T3 ES18827388T ES18827388T ES2958662T3 ES 2958662 T3 ES2958662 T3 ES 2958662T3 ES 18827388 T ES18827388 T ES 18827388T ES 18827388 T ES18827388 T ES 18827388T ES 2958662 T3 ES2958662 T3 ES 2958662T3
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rsk3
composition
makap
cardiac
mice
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Michael S Kapiloff
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
    • A01K67/0276—Knock-out vertebrates
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • 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
    • A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • 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
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    • 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
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    • 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
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    • 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
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
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    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
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    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
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    • 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
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cardiology (AREA)
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  • Virology (AREA)
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ES18827388T 2017-07-06 2018-07-05 Tratamiento de cardiopatía por inhibición de la acción de la proteína de anclaje a la cinasa A del músculo (mAKAP) Active ES2958662T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762529224P 2017-07-06 2017-07-06
PCT/US2018/040913 WO2019010301A1 (en) 2017-07-06 2018-07-05 TREATMENT OF CARDIOPATHIES BY INHIBITING THE ACTION OF PROTEINS FROM ANCHORING TO MUSCLE PROTEIN KINASES A (MAKAP)

Publications (1)

Publication Number Publication Date
ES2958662T3 true ES2958662T3 (es) 2024-02-13

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ES18827388T Active ES2958662T3 (es) 2017-07-06 2018-07-05 Tratamiento de cardiopatía por inhibición de la acción de la proteína de anclaje a la cinasa A del músculo (mAKAP)

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US (1) US10907153B2 (enExample)
EP (1) EP3658157B1 (enExample)
JP (1) JP7193874B2 (enExample)
AU (1) AU2018297274B2 (enExample)
CA (1) CA3065816C (enExample)
ES (1) ES2958662T3 (enExample)
WO (1) WO2019010301A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9132174B2 (en) 2013-03-15 2015-09-15 Anchored Rsk3 Inhibitors, Llc Treatment of heart disease by inhibition of the action of ribosomal S6 kinase 3 (RSK3)
EP3846843A4 (en) * 2018-09-06 2022-06-22 Icahn School of Medicine at Mount Sinai COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEART DISEASES
SG11202112499VA (en) * 2019-05-15 2021-12-30 Univ Miami Treatment of heart disease by disruption of the anchoring of pp2a
FI4103214T3 (fi) 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
CN120677240A (zh) * 2023-02-13 2025-09-19 科瑞生物科技股份有限公司 人肌钙蛋白t基因的启动子-增强子序列用于心肌细胞中的选择性表达

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6489125B1 (en) 2000-05-10 2002-12-03 The Trustees Of Columbia University In The City Of New York Methods for identifying chemical compounds that inhibit dissociation of FKBP12.6 binding protein from type 2 ryanodine receptor
EP2305813A3 (en) * 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
BRPI0410787A (pt) 2003-05-21 2006-06-20 Univ Texas inibição da proteìna cinase c-mu (pkd) como um tratamento para hipertrofia cardìaca e insuficiência cardìaca
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) * 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
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
US9132174B2 (en) 2013-03-15 2015-09-15 Anchored Rsk3 Inhibitors, Llc Treatment of heart disease by inhibition of the action of ribosomal S6 kinase 3 (RSK3)

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CA3065816C (en) 2024-11-19
WO2019010301A1 (en) 2019-01-10
JP2020527167A (ja) 2020-09-03
EP3658157A4 (en) 2021-03-24
AU2018297274A1 (en) 2020-01-23
EP3658157A1 (en) 2020-06-03
EP3658157B1 (en) 2023-07-26
CA3065816A1 (en) 2019-01-10
JP7193874B2 (ja) 2022-12-21
US10907153B2 (en) 2021-02-02
WO2019010301A8 (en) 2020-05-22
US20190010493A1 (en) 2019-01-10
AU2018297274B2 (en) 2023-05-18

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