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MX2024004386A - Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso. - Google Patents

Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.

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Publication number
MX2024004386A
MX2024004386A MX2024004386A MX2024004386A MX2024004386A MX 2024004386 A MX2024004386 A MX 2024004386A MX 2024004386 A MX2024004386 A MX 2024004386A MX 2024004386 A MX2024004386 A MX 2024004386A MX 2024004386 A MX2024004386 A MX 2024004386A
Authority
MX
Mexico
Prior art keywords
gene
correction
methods
therapeutic targets
human dystrophin
Prior art date
Application number
MX2024004386A
Other languages
English (en)
Inventor
Charles A Gersbach
Jacqueline N Robinson-Hamm
Original Assignee
Univ Duke
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Application filed by Univ Duke filed Critical Univ Duke
Publication of MX2024004386A publication Critical patent/MX2024004386A/es

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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
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    • A61K47/51Medicinal 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 non-active ingredient being a modifying agent
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • A61K48/0016Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
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    • A61K48/005Medicinal 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
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Abstract

En este documento se divulgan dianas terapéuticas para la corrección del gen de la distrofina humana por edición génica y métodos de uso.
MX2024004386A 2015-11-30 2018-04-26 Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso. MX2024004386A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562260712P 2015-11-30 2015-11-30
US201662330336P 2016-05-02 2016-05-02

Publications (1)

Publication Number Publication Date
MX2024004386A true MX2024004386A (es) 2024-04-29

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MX2018005377A MX2018005377A (es) 2015-11-30 2016-11-30 Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.
MX2024004386A MX2024004386A (es) 2015-11-30 2018-04-26 Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.

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Country Link
US (1) US12214054B2 (es)
EP (1) EP3384055A4 (es)
JP (3) JP7108307B2 (es)
KR (1) KR20180081618A (es)
CN (2) CN118147141A (es)
AU (1) AU2016362282B2 (es)
CA (1) CA3001623A1 (es)
EA (1) EA201891317A3 (es)
IL (1) IL259100B2 (es)
MX (2) MX2018005377A (es)
WO (1) WO2017095967A2 (es)

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EP4177346A3 (en) 2015-08-25 2023-07-26 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
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WO2017193029A2 (en) * 2016-05-05 2017-11-09 Duke University Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy
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JOP20190166A1 (ar) * 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
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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WO2020210776A1 (en) * 2019-04-12 2020-10-15 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
AR118670A1 (es) * 2019-04-14 2021-10-20 Univ Duke Eliminación mediada por vectores aav de grandes puntos de mutación para el tratamiento de la distrofia muscular de duchenne
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US20240091379A1 (en) * 2019-10-11 2024-03-21 Yale University Compositions and methods for upregulating isoforms of dystrophin as therapy for duchenne muscular dystrophy (dmd)
US20230287389A1 (en) * 2019-11-07 2023-09-14 Qingdao Kingagroot Chemical Compound Co., Ltd. A method for generating new mutation in organism and use thereof
WO2021222314A1 (en) * 2020-04-27 2021-11-04 Duke University Gene editing of satellite cells in vivo using aav vectors encoding muscle-specific promoters
US20230349888A1 (en) * 2020-04-27 2023-11-02 Duke University A high-throughput screening method to discover optimal grna pairs for crispr-mediated exon deletion
CN112522256B (zh) * 2020-08-19 2023-08-22 南京启真基因工程有限公司 CRISPR/Cas9系统及其在构建抗肌萎缩蛋白基因缺陷的猪源重组细胞中的应用
JP2023545132A (ja) * 2020-10-12 2023-10-26 デューク ユニバーシティ ジストロフィン機能を修復するためのcrispr/casをベースにした塩基編集組成物
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US20180353615A1 (en) 2018-12-13
AU2016362282A1 (en) 2018-05-10
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CN118147141A (zh) 2024-06-07
EA201891317A3 (ru) 2019-04-30
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JP2022160426A (ja) 2022-10-19
JP2018534950A (ja) 2018-11-29
BR112018011133A2 (pt) 2018-11-21
IL259100B2 (en) 2023-09-01
JP2024153629A (ja) 2024-10-29
CA3001623A1 (en) 2017-06-08
CN108779466B (zh) 2024-03-29
EP3384055A2 (en) 2018-10-10
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IL259100A (en) 2018-06-28
AU2016362282B2 (en) 2023-03-16
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EP3384055A4 (en) 2019-04-17
EA201891317A2 (ru) 2018-12-28

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