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MX2024013018A - 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

Info

Publication number
MX2024013018A
MX2024013018A MX2024013018A MX2024013018A MX2024013018A MX 2024013018 A MX2024013018 A MX 2024013018A MX 2024013018 A MX2024013018 A MX 2024013018A MX 2024013018 A MX2024013018 A MX 2024013018A MX 2024013018 A MX2024013018 A MX 2024013018A
Authority
MX
Mexico
Prior art keywords
gene
correction
methods
therapeutic targets
human dystrophin
Prior art date
Application number
MX2024013018A
Other languages
English (en)
Inventor
Charles A Gersbach
Hamm Jacqueline N Robinson-
Original Assignee
Univ Duke
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 Duke filed Critical Univ Duke
Publication of MX2024013018A publication Critical patent/MX2024013018A/es

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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
    • C12N15/11—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K47/51—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 non-active ingredient being a modifying agent
    • A61K47/54—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 non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • 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
    • A61K48/0008—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 '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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    • A61K48/0008—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 '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
    • A61K48/0016—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 '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
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
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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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    • C12N15/00—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
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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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
    • C12N15/63—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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
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    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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  • Health & Medical Sciences (AREA)
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  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Environmental Sciences (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
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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.
MX2024013018A 2015-11-30 2018-04-26 Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso MX2024013018A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562260712P 2015-11-30 2015-11-30
US201662330336P 2016-05-02 2016-05-02
PCT/US2016/064285 WO2017095967A2 (en) 2015-11-30 2016-11-30 Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use

Publications (1)

Publication Number Publication Date
MX2024013018A true MX2024013018A (es) 2024-11-08

Family

ID=58797730

Family Applications (3)

Application Number Title Priority Date Filing Date
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.
MX2024013018A MX2024013018A (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

Family Applications Before (2)

Application Number Title Priority Date Filing Date
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.

Country Status (11)

Country Link
US (1) US12214054B2 (es)
EP (2) EP4644567A2 (es)
JP (3) JP7108307B2 (es)
KR (2) KR20250044471A (es)
CN (2) CN108779466B (es)
AU (1) AU2016362282B2 (es)
CA (1) CA3001623A1 (es)
EA (1) EA201891317A3 (es)
IL (1) IL259100B2 (es)
MX (3) MX2018005377A (es)
WO (1) WO2017095967A2 (es)

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EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
ES2929110T3 (es) 2015-08-25 2022-11-24 Univ Duke Composiciones y métodos para mejorar la especificidad en ingeniería genética usando endonucleasas guiadas por ARN
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
DK4279084T3 (da) 2015-10-28 2025-09-15 Vertex Pharma Materialer og fremgangsmåder til behandling af duchennes muskeldystrofi
EP4644567A2 (en) * 2015-11-30 2025-11-05 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2017193029A2 (en) * 2016-05-05 2017-11-09 Duke University Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
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
WO2019036599A1 (en) * 2017-08-18 2019-02-21 The Board Of Regents Of The University Of Texas System EXON DELETION CORRECTION OF MUTATIONS OF DUCHENNE MUSCLE DYSTROPHY IN ACTINE DYSTROPHINE BINDING DOMAIN 1 Using a GENOME CRISPR EDITION
MA50578A (fr) * 2017-11-09 2021-09-15 Vertex Pharma Systèmes crispr/cas pour le traitement de dmd
US11891635B2 (en) 2017-12-21 2024-02-06 Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft Nucleic acid sequence replacement by NHEJ
EP3768303B1 (en) * 2018-03-23 2024-12-18 Massachusetts Eye and Ear Infirmary Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome
WO2019209777A1 (en) 2018-04-23 2019-10-31 The Curators Of The University Of Missouri Improved crispr therapy
CN113646004A (zh) * 2018-12-12 2021-11-12 坚固生物科技公司 用于治疗肌营养不良的组合疗法
US20220177879A1 (en) * 2019-04-12 2022-06-09 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
US20220184229A1 (en) * 2019-04-14 2022-06-16 Duke University Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
US20210047649A1 (en) * 2019-05-08 2021-02-18 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
CN110499333A (zh) * 2019-08-01 2019-11-26 广州德赫生物科技有限公司 用于修复dmd基因突变的核酸序列及系统
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