HK1252157A1 - 重組病毒產品和抑制dux4表達的方法 - Google Patents
重組病毒產品和抑制dux4表達的方法Info
- Publication number
- HK1252157A1 HK1252157A1 HK18111550.9A HK18111550A HK1252157A1 HK 1252157 A1 HK1252157 A1 HK 1252157A1 HK 18111550 A HK18111550 A HK 18111550A HK 1252157 A1 HK1252157 A1 HK 1252157A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- dux4
- inhibition
- expression
- methods
- recombinant virus
- Prior art date
Links
Classifications
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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
- C12N15/86—Viral vectors
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- 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
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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
- C12N15/11—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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- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
- C12N2750/00032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- General Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161511319P | 2011-07-25 | 2011-07-25 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HK1252157A1 true HK1252157A1 (zh) | 2019-05-17 |
Family
ID=47601499
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HK18111550.9A HK1252157A1 (zh) | 2011-07-25 | 2018-09-07 | 重組病毒產品和抑制dux4表達的方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (3) | US9469851B2 (zh) |
| EP (2) | EP3290055A3 (zh) |
| AU (4) | AU2012286994B2 (zh) |
| CA (2) | CA3106285A1 (zh) |
| ES (1) | ES2648169T3 (zh) |
| HK (1) | HK1252157A1 (zh) |
| WO (1) | WO2013016352A1 (zh) |
Families Citing this family (79)
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| US10865445B2 (en) * | 2010-08-18 | 2020-12-15 | Fred Hutchinson Cancer Research Center | Methods for alleviating facioscapulohumeral dystrophy (FSHD) by N siRNA molecule inhibiting the expression of DUX4-FL |
| US20120225034A1 (en) | 2010-09-02 | 2012-09-06 | Universite De Mons | Agents useful in treating facioscapulohumeral muscular dystrophy |
| EP3290055A3 (en) * | 2011-07-25 | 2018-05-30 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of dux4 |
| RU2708032C2 (ru) | 2013-02-20 | 2019-12-03 | Новартис Аг | ЛЕЧЕНИЕ РАКА С ИСПОЛЬЗОВАНИЕМ ХИМЕРНОГО АНТИГЕНСПЕЦИФИЧЕСКОГО РЕЦЕПТОРА НА ОСНОВЕ ГУМАНИЗИРОВАННОГО АНТИТЕЛА ПРОТИВ EGFRvIII |
| TW201446794A (zh) | 2013-02-20 | 2014-12-16 | Novartis Ag | 利用抗－ｃｄ１２３嵌合抗原受體工程化ｔ細胞之初級人類白血病有效靶向 |
| EP2970426B1 (en) | 2013-03-15 | 2019-08-28 | Michael C. Milone | Targeting cytotoxic cells with chimeric receptors for adoptive immunotherapy |
| UY35468A (es) | 2013-03-16 | 2014-10-31 | Novartis Ag | Tratamiento de cáncer utilizando un receptor quimérico de antígeno anti-cd19 |
| KR20220119187A (ko) * | 2013-05-15 | 2022-08-26 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
| US10640569B2 (en) | 2013-12-19 | 2020-05-05 | Novartis Ag | Human mesothelin chimeric antigen receptors and uses thereof |
| EP3087101B1 (en) | 2013-12-20 | 2024-06-05 | Novartis AG | Regulatable chimeric antigen receptor |
| WO2015112626A1 (en) | 2014-01-21 | 2015-07-30 | June Carl H | Enhanced antigen presenting ability of car t cells by co-introduction of costimulatory molecules |
| US10538809B2 (en) * | 2014-01-28 | 2020-01-21 | Qiagen Gmbh | Method of amplification of a short tandem repeat locus |
| IL280215B (en) | 2014-04-07 | 2022-07-01 | Novartis Ag | Cancer treatment using a chimeric receptor antigen (car) against cd19 |
| JP6831777B2 (ja) | 2014-07-21 | 2021-02-17 | ノバルティス アーゲー | Ｃｄ３３キメラ抗原受容体を使用する癌の処置 |
| RU2741120C2 (ru) | 2014-07-21 | 2021-01-22 | Новартис Аг | Лечение рака с использованием химерного антигенного рецептора cll-1 |
| WO2016014553A1 (en) | 2014-07-21 | 2016-01-28 | Novartis Ag | Sortase synthesized chimeric antigen receptors |
| RU2751660C2 (ru) | 2014-07-21 | 2021-07-15 | Новартис Аг | Лечение злокачественного новообразования с использованием гуманизированного химерного антигенного рецептора против всма |
| CN107108744B (zh) | 2014-08-19 | 2020-09-25 | 诺华股份有限公司 | 抗cd123嵌合抗原受体(car)用于癌症治疗 |
| CA2961636A1 (en) | 2014-09-17 | 2016-03-24 | Boris ENGELS | Targeting cytotoxic cells with chimeric receptors for adoptive immunotherapy |
| CN106973568B (zh) | 2014-10-08 | 2021-07-23 | 诺华股份有限公司 | 预测针对嵌合抗原受体疗法的治疗应答性的生物标志及其用途 |
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| SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
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| MA47800A (fr) | 2017-03-17 | 2020-01-22 | Univ Newcastle | Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire |
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| EP4186919A1 (en) | 2021-11-30 | 2023-05-31 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| WO2024064913A1 (en) | 2022-09-23 | 2024-03-28 | Sarepta Therapeutics, Inc. | Recombinant aav vectors for treating muscular dystrophy |
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2012
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- 2012-07-24 WO PCT/US2012/047999 patent/WO2013016352A1/en active Application Filing
- 2012-07-24 CA CA3106285A patent/CA3106285A1/en active Pending
- 2012-07-24 CA CA2842798A patent/CA2842798C/en active Active
- 2012-07-24 AU AU2012286994A patent/AU2012286994B2/en active Active
- 2012-07-24 US US14/234,413 patent/US9469851B2/en active Active
- 2012-07-24 ES ES12816988.5T patent/ES2648169T3/es active Active
- 2012-07-24 EP EP12816988.5A patent/EP2736539B1/en active Active
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2016
- 2016-10-17 US US15/295,591 patent/US10301649B2/en active Active
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2017
- 2017-11-10 AU AU2017258945A patent/AU2017258945A1/en not_active Abandoned
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2018
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2019
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2022
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| ES2648169T3 (es) | 2017-12-28 |
| US20140322169A1 (en) | 2014-10-30 |
| US10301649B2 (en) | 2019-05-28 |
| AU2012286994A1 (en) | 2014-02-13 |
| WO2013016352A1 (en) | 2013-01-31 |
| CA3106285A1 (en) | 2013-01-31 |
| AU2012286994B2 (en) | 2017-08-10 |
| EP3290055A2 (en) | 2018-03-07 |
| WO2013016352A8 (en) | 2013-09-12 |
| AU2020200323A1 (en) | 2020-02-06 |
| EP2736539A4 (en) | 2015-04-08 |
| US20170029849A1 (en) | 2017-02-02 |
| US11802291B2 (en) | 2023-10-31 |
| CA2842798C (en) | 2021-02-23 |
| CA2842798A1 (en) | 2013-01-31 |
| US9469851B2 (en) | 2016-10-18 |
| AU2017258945A1 (en) | 2018-01-04 |
| EP2736539A1 (en) | 2014-06-04 |
| AU2022201820A1 (en) | 2022-04-07 |
| EP2736539B1 (en) | 2017-08-23 |
| EP3290055A3 (en) | 2018-05-30 |
| US20190300903A1 (en) | 2019-10-03 |
| AU2020200323B2 (en) | 2022-03-31 |
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