MX2018005377A - 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
- MX2018005377A MX2018005377A MX2018005377A MX2018005377A MX2018005377A MX 2018005377 A MX2018005377 A MX 2018005377A MX 2018005377 A MX2018005377 A MX 2018005377A MX 2018005377 A MX2018005377 A MX 2018005377A MX 2018005377 A MX2018005377 A MX 2018005377A
- Authority
- MX
- Mexico
- Prior art keywords
- gene
- correction
- methods
- therapeutic targets
- human dystrophin
- Prior art date
Links
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 title abstract 2
- 238000010362 genome editing Methods 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 230000001225 therapeutic effect Effects 0.000 title abstract 2
Classifications
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- 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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
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- 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
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- 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
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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
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- A—HUMAN NECESSITIES
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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
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- 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
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- C12N15/102—Mutagenizing nucleic acids
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- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/80—Vectors or expression systems specially adapted for eukaryotic hosts for fungi
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- 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
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
- C12N9/22—Ribonucleases RNAses, DNAses
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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- 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
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
- C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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- 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
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- 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
Abstract
En este documento se divulgan dianas terapéuticas para la corrección del gen de la distrofia humana por edición génica y métodos de uso.
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 |
---|---|
MX2018005377A true MX2018005377A (es) | 2018-11-09 |
Family
ID=58797730
Family Applications (1)
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. |
Country Status (12)
Country | Link |
---|---|
US (1) | US20180353615A1 (es) |
EP (1) | EP3384055A4 (es) |
JP (2) | JP7108307B2 (es) |
KR (1) | KR20180081618A (es) |
CN (1) | CN108779466B (es) |
AU (1) | AU2016362282B2 (es) |
BR (1) | BR112018011133A2 (es) |
CA (1) | CA3001623A1 (es) |
EA (1) | EA201891317A3 (es) |
IL (1) | IL259100B2 (es) |
MX (1) | MX2018005377A (es) |
WO (1) | WO2017095967A2 (es) |
Families Citing this family (23)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | Duke University | Genetic correction of mutated genes |
EP3256487A4 (en) | 2015-02-09 | 2018-07-18 | 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 |
US11970710B2 (en) | 2015-10-13 | 2024-04-30 | Duke University | Genome engineering with Type I CRISPR systems in eukaryotic cells |
US11369692B2 (en) | 2015-10-28 | 2022-06-28 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of Duchenne Muscular Dystrophy |
ES2957660T3 (es) * | 2016-05-05 | 2024-01-23 | Univ Duke | Composiciones relacionadas con crispr/cas para tratar la distrofia muscular de duchenne |
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 |
EP3668983A1 (en) * | 2017-08-18 | 2020-06-24 | The Board of Regents of The University of Texas System | Exon deletion correction of duchenne muscular dystrophy mutations in the dystrophin actin binding domain 1 using crispr genome editing |
MA50578A (fr) * | 2017-11-09 | 2021-09-15 | Vertex Pharma | Systèmes crispr/cas pour le traitement de dmd |
WO2019122302A1 (en) | 2017-12-21 | 2019-06-27 | Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft | Nucleic acid sequence replacement by nhej |
WO2019183641A1 (en) * | 2018-03-23 | 2019-09-26 | Massachusetts Eye And Ear Infirmary | Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome |
EP3893940A4 (en) * | 2018-12-12 | 2022-09-28 | Solidus Biosciences, Inc. | POLYTHERAPY FOR THE TREATMENT OF MUSCULAR DYSTROPHY |
JP2022526669A (ja) * | 2019-04-12 | 2022-05-25 | 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 |
TW202104594A (zh) * | 2019-04-14 | 2021-02-01 | 美國公爵大學 | 治療杜興氏(duchenne)肌肉失養症之大型突變熱點之aav載體介導之刪除 |
WO2020225606A1 (en) * | 2019-05-08 | 2020-11-12 | Crispr Therapeutics Ag | 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*** |
WO2021072276A1 (en) * | 2019-10-11 | 2021-04-15 | Yale University | Compositions and methods for upregulating isoforms of dystrophin as therapy for duchenne muscular dystrophy (dmd) |
KR20220093324A (ko) * | 2019-11-07 | 2022-07-05 | 칭다오 킹아그루트 케미컬 컴파운드 컴퍼니 리미티드 | 유기체에서 새로운 돌연변이를 발생시키는 방법 및 그 활용 |
US20230348870A1 (en) * | 2020-04-27 | 2023-11-02 | Duke University | Gene editing of satellite cells in vivo using aav vectors encoding muscle-specific promoters |
CN112522256B (zh) * | 2020-08-19 | 2023-08-22 | 南京启真基因工程有限公司 | CRISPR/Cas9***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 |
EP4225907A1 (en) * | 2020-10-12 | 2023-08-16 | Duke University | Crispr/cas-based base editing composition for restoring dystrophin function |
EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
Family Cites Families (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB9011454D0 (en) * | 1990-05-22 | 1990-07-11 | Medical Res Council | Polynucleotide amplification |
AU4528493A (en) | 1992-06-04 | 1994-01-04 | Regents Of The University Of California, The | In vivo gene therapy with intron-free sequence of interest |
ATE302854T1 (de) | 1993-01-26 | 2005-09-15 | Univ Pennsylvania | Zusammensetzungen und verfahren zur verabreichung von genetischem material |
US5593972A (en) | 1993-01-26 | 1997-01-14 | The Wistar Institute | Genetic immunization |
US5962428A (en) | 1995-03-30 | 1999-10-05 | Apollon, Inc. | Compositions and methods for delivery of genetic material |
US20040192593A1 (en) | 1999-07-26 | 2004-09-30 | Baylor College Of Medicine | Protease resistant ti-growth hormone releasing hormone |
AU2003295366B2 (en) | 2002-11-04 | 2011-11-24 | Advisys, Inc. | Synthetic muscle promoters with activities exceeding naturally occurring regulatory sequences in cardiac cells |
CA2906812A1 (en) * | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
EP3417880A1 (en) * | 2013-06-05 | 2018-12-26 | Duke University | Rna-guided gene editing and gene regulation |
EP3277816B1 (en) * | 2015-04-01 | 2020-06-17 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy |
ES2957660T3 (es) * | 2016-05-05 | 2024-01-23 | Univ Duke | Composiciones relacionadas con crispr/cas para tratar la distrofia muscular de duchenne |
-
2016
- 2016-11-30 JP JP2018547872A patent/JP7108307B2/ja active Active
- 2016-11-30 KR KR1020187018218A patent/KR20180081618A/ko not_active Application Discontinuation
- 2016-11-30 CN CN201680080439.7A patent/CN108779466B/zh active Active
- 2016-11-30 EA EA201891317A patent/EA201891317A3/ru unknown
- 2016-11-30 CA CA3001623A patent/CA3001623A1/en active Pending
- 2016-11-30 EP EP16871452.5A patent/EP3384055A4/en active Pending
- 2016-11-30 BR BR112018011133A patent/BR112018011133A2/pt active Search and Examination
- 2016-11-30 AU AU2016362282A patent/AU2016362282B2/en active Active
- 2016-11-30 IL IL259100A patent/IL259100B2/en unknown
- 2016-11-30 WO PCT/US2016/064285 patent/WO2017095967A2/en active Application Filing
- 2016-11-30 MX MX2018005377A patent/MX2018005377A/es unknown
- 2016-11-30 US US15/779,633 patent/US20180353615A1/en active Pending
-
2022
- 2022-07-08 JP JP2022110179A patent/JP2022160426A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
IL259100B1 (en) | 2023-05-01 |
US20180353615A1 (en) | 2018-12-13 |
EA201891317A3 (ru) | 2019-04-30 |
BR112018011133A2 (pt) | 2018-11-21 |
JP7108307B2 (ja) | 2022-07-28 |
KR20180081618A (ko) | 2018-07-16 |
IL259100B2 (en) | 2023-09-01 |
WO2017095967A2 (en) | 2017-06-08 |
EP3384055A2 (en) | 2018-10-10 |
IL259100A (en) | 2018-06-28 |
JP2022160426A (ja) | 2022-10-19 |
CN108779466A (zh) | 2018-11-09 |
CN108779466B (zh) | 2024-03-29 |
AU2016362282A1 (en) | 2018-05-10 |
JP2018534950A (ja) | 2018-11-29 |
AU2016362282B2 (en) | 2023-03-16 |
EP3384055A4 (en) | 2019-04-17 |
EA201891317A2 (ru) | 2018-12-28 |
CA3001623A1 (en) | 2017-06-08 |
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