IL276139A - Compositions and methods for correcting dystrophin mutations in human cardiomyocytes - Google Patents

Compositions and methods for correcting dystrophin mutations in human cardiomyocytes

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Publication number
IL276139A
IL276139A IL276139A IL27613920A IL276139A IL 276139 A IL276139 A IL 276139A IL 276139 A IL276139 A IL 276139A IL 27613920 A IL27613920 A IL 27613920A IL 276139 A IL276139 A IL 276139A
Authority
IL
Israel
Prior art keywords
compositions
methods
human cardiomyocytes
dystrophin mutations
correcting
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Application number
IL276139A
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English (en)
Hebrew (he)
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Univ Texas
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Publication date
Application filed by Univ Texas filed Critical Univ Texas
Publication of IL276139A publication Critical patent/IL276139A/en

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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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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
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/34—Muscles; Smooth muscle cells; Heart; Cardiac stem cells; Myoblasts; Myocytes; Cardiomyocytes
    • A—HUMAN NECESSITIES
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
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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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
IL276139A 2018-01-31 2020-07-19 Compositions and methods for correcting dystrophin mutations in human cardiomyocytes IL276139A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862624748P 2018-01-31 2018-01-31
PCT/US2019/015988 WO2019152609A1 (en) 2018-01-31 2019-01-31 Compositions and methods for correcting dystrophin mutations in human cardiomyocytes

Publications (1)

Publication Number Publication Date
IL276139A true IL276139A (en) 2020-09-30

Family

ID=65657512

Family Applications (1)

Application Number Title Priority Date Filing Date
IL276139A IL276139A (en) 2018-01-31 2020-07-19 Compositions and methods for correcting dystrophin mutations in human cardiomyocytes

Country Status (11)

Country Link
US (1) US20200370042A1 (https=)
EP (1) EP3746557A1 (https=)
JP (1) JP2021511803A (https=)
KR (1) KR20200116933A (https=)
CN (1) CN111836893A (https=)
AU (1) AU2019216321A1 (https=)
BR (1) BR112020015617A2 (https=)
CA (1) CA3088547A1 (https=)
EA (1) EA202091828A1 (https=)
IL (1) IL276139A (https=)
WO (1) WO2019152609A1 (https=)

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US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
AR118668A1 (es) * 2019-04-14 2021-10-20 Univ Duke Composiciones para editar genoma a base de crispr / cas para restaurar la función de distrofina
WO2021064162A1 (en) * 2019-10-02 2021-04-08 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Treatment of diseases caused by frame shift mutations
EP4069314A4 (en) * 2019-12-03 2024-06-26 Duke University SYSTEMS AND METHODS FOR LIPID NANOPARTICLE DELIVERY OF GENE EDITING MACHINERY
US20230272428A1 (en) * 2019-12-16 2023-08-31 President And Fellows Of Harvard College Methods and compositions for correction of dmd mutations
CN115011598A (zh) * 2020-09-02 2022-09-06 西湖大学 杜氏肌营养不良症相关的外显子剪接增强子、sgRNA、基因编辑工具及应用
US20240254509A1 (en) * 2021-03-04 2024-08-01 Research Institute At Nationwide Children's Hospital Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
AU2022262420A1 (en) * 2021-04-23 2023-11-16 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2023039444A2 (en) * 2021-09-08 2023-03-16 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exon 51 for treatment of duchenne muscular dystrophy
WO2023172926A1 (en) * 2022-03-08 2023-09-14 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exons for treatment of duchenne muscular dystrophy
WO2023172927A1 (en) * 2022-03-08 2023-09-14 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exon 44, 50, and 53 for treatment of duchenne muscular dystrophy
KR20230134098A (ko) * 2022-03-10 2023-09-20 주식회사 진코어 듀센 근이영양증 치료를 위한 유전자 편집 시스템 및 이를 이용한 질병 치료 방법
WO2023240157A2 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd

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BR122020021379B1 (pt) * 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
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JP7075597B2 (ja) * 2016-05-05 2022-05-26 デューク ユニバーシティ デュシェンヌ型筋ジストロフィーを治療するためのｃｒｉｓｐｒ／ｃａｓ関連の方法および組成物
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JP7490211B2 (ja) * 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
RU2625003C1 (ru) * 2016-10-04 2017-07-11 Федеральное государственное бюджетное научное учреждение "Медико-генетический научный центр" Набор последовательностей нуклеотидов для медицинской технологии детекции наиболее частых в россии делеций гена dmd методом мультиплексного пцр/пдаф анализа
US10687520B2 (en) * 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44

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Publication number Publication date
KR20200116933A (ko) 2020-10-13
US20200370042A1 (en) 2020-11-26
BR112020015617A2 (pt) 2021-01-05
CN111836893A (zh) 2020-10-27
EA202091828A1 (ru) 2021-05-24
WO2019152609A1 (en) 2019-08-08
EP3746557A1 (en) 2020-12-09
CA3088547A1 (en) 2019-08-08
AU2019216321A1 (en) 2020-07-30
JP2021511803A (ja) 2021-05-13

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