MX2023001600A - Variantes de factor viii reducido en cpg y composiciones para usarse en el tratamiento de trastornos de hemostasia. - Google Patents
Variantes de factor viii reducido en cpg y composiciones para usarse en el tratamiento de trastornos de hemostasia.Info
- Publication number
- MX2023001600A MX2023001600A MX2023001600A MX2023001600A MX2023001600A MX 2023001600 A MX2023001600 A MX 2023001600A MX 2023001600 A MX2023001600 A MX 2023001600A MX 2023001600 A MX2023001600 A MX 2023001600A MX 2023001600 A MX2023001600 A MX 2023001600A
- Authority
- MX
- Mexico
- Prior art keywords
- factor viii
- methods
- cpg reduced
- compositions
- treatment
- Prior art date
Links
- 229960000301 factor viii Drugs 0.000 title 1
- 230000023597 hemostasis Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 102100026735 Coagulation factor VIII Human genes 0.000 abstract 2
- 108010054218 Factor VIII Proteins 0.000 abstract 2
- 102000001690 Factor VIII Human genes 0.000 abstract 2
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 230000000694 effects Effects 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
Classifications
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- 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
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
- A61K38/37—Factors VIII
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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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
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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
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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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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- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10342—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- 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
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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
- C12N2750/14145—Special targeting system for viral vectors
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
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- Diabetes (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)
- Peptides Or Proteins (AREA)
Abstract
Se dan a conocer variantes de ácido nucleico reducido en CpG que codifican la proteína FVIII, y métodos de uso de las mismas. En modalidades particulares, las variantes de ácido nucleico reducido en CpG que codifican FVIII se expresan con mayor eficiencia por las células, se secretan en niveles incrementados por las células por encima de las proteínas Factor VIII de tipo silvestre, exhiben una expresión y/o actividad potenciadas sobre las proteínas Factor VIII de tipo silvestre, o se empaquetan con más eficiencia en vectores virales.
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562249001P | 2015-10-30 | 2015-10-30 | |
| US201662331872P | 2016-05-04 | 2016-05-04 | |
| US201662349532P | 2016-06-13 | 2016-06-13 | |
| US201662357874P | 2016-07-01 | 2016-07-01 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023001600A true MX2023001600A (es) | 2023-03-09 |
Family
ID=58630834
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2018005490A MX2018005490A (es) | 2015-10-30 | 2016-10-31 | Variantes de factor viii reducido en cpg, composiciones, metodos y usos para el tratamiento de trastornos de hemostasia. |
| MX2023001600A MX2023001600A (es) | 2015-10-30 | 2018-04-30 | Variantes de factor viii reducido en cpg y composiciones para usarse en el tratamiento de trastornos de hemostasia. |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2018005490A MX2018005490A (es) | 2015-10-30 | 2016-10-31 | Variantes de factor viii reducido en cpg, composiciones, metodos y usos para el tratamiento de trastornos de hemostasia. |
Country Status (19)
| Country | Link |
|---|---|
| US (3) | US20180312571A1 (es) |
| EP (1) | EP3368560A4 (es) |
| JP (3) | JP7088831B2 (es) |
| KR (1) | KR20180090795A (es) |
| CN (1) | CN108368164A (es) |
| AU (1) | AU2016344030B2 (es) |
| BR (1) | BR112018008766A2 (es) |
| CA (1) | CA3002689A1 (es) |
| CL (1) | CL2018001107A1 (es) |
| CO (1) | CO2018005306A2 (es) |
| IL (1) | IL258959B2 (es) |
| MX (2) | MX2018005490A (es) |
| MY (1) | MY190926A (es) |
| PE (2) | PE20231949A1 (es) |
| PH (1) | PH12018501055A1 (es) |
| RU (1) | RU2745506C2 (es) |
| SA (1) | SA518391463B1 (es) |
| SG (1) | SG11201802972UA (es) |
| WO (1) | WO2017075619A1 (es) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| PL3313991T3 (pl) | 2015-06-23 | 2024-11-04 | The Children's Hospital Of Philadelphia | Modyfikowany czynnik ix oraz kompozycje, sposoby i zastosowania do transferu genów do komórek, narządów i tkanek |
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| EP3491008A2 (en) | 2016-07-26 | 2019-06-05 | BioMarin Pharmaceutical Inc. | Novel adeno-associated virus capsid proteins |
| JP7499174B2 (ja) * | 2017-08-01 | 2024-06-13 | スパーク セラピューティクス インコーポレイテッド | 第viii因子(fviii)遺伝子治療法 |
| IL322212A (en) * | 2017-08-09 | 2025-09-01 | Bioverativ Therapeutics Inc | Nucleic acid molecules and their uses |
| KR101952102B1 (ko) * | 2017-12-07 | 2019-02-26 | 주식회사 지앤피바이오사이언스 | 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터 |
| AU2019265560A1 (en) | 2018-05-09 | 2020-11-26 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| EP3794112A1 (en) | 2018-05-14 | 2021-03-24 | BioMarin Pharmaceutical Inc. | Stable expression of aav vectors in juvenile subjects |
| BR112021002017A2 (pt) | 2018-08-09 | 2021-05-11 | Bioverativ Therapeutics Inc. | moléculas de ácido nucleico e usos das mesmas para terapia genética não viral |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| BR112021003399A2 (pt) * | 2018-08-24 | 2021-05-18 | Spark Therapeutics, Inc. | sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso |
| CA3119349A1 (en) * | 2018-11-16 | 2020-05-22 | Spark Therapeutics, Inc. | In vitro assay for detecting enhancers and inhibitors of adeno associated virus (aav) vector transduction and/or detecting or quantitating anti-aav binding antibodies |
| GB201900702D0 (en) * | 2019-01-18 | 2019-03-06 | Univ Bristol | Therapy |
| CA3129532A1 (en) | 2019-02-15 | 2020-08-20 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| MX2021011039A (es) | 2019-03-13 | 2021-12-15 | Generation Bio Co | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con fviii. |
| CN114901686A (zh) * | 2019-11-01 | 2022-08-12 | 自由行疗法有限公司 | 因子viii多肽 |
| EP4090382A2 (en) * | 2020-01-16 | 2022-11-23 | Takeda Pharmaceutical Company Limited | Adeno associated virus based gene therapy for phenylketonuria |
| GB202009039D0 (en) | 2020-06-15 | 2020-07-29 | Univ Bristol | Therapy |
| WO2023036054A2 (en) * | 2021-09-08 | 2023-03-16 | Inspirar Limited | Composition and method for treating hemophilia |
| CR20240524A (es) * | 2022-04-28 | 2025-05-05 | Biocad Joint Stock Co | Ácido nucleico de codón optimizado que codifica la proteína del factor viii de coagulación de dominio b suprimido, y uso del mismo |
| CN120166940A (zh) * | 2022-08-31 | 2025-06-17 | 流体基因治疗有限公司 | 流体动力学基因递送的方法和组合物 |
| CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
| WO2025164792A1 (ja) * | 2024-02-02 | 2025-08-07 | 学校法人自治医科大学 | 高比活性/高分泌発現型の第viii因子改変体、高発現型の第viii因子コード核酸及びそれらの用途 |
| WO2025180508A1 (en) * | 2024-02-29 | 2025-09-04 | Shanghai Vitalgen Biopharma Co., Ltd. | Human factor viii variant expression cassettes and uses thereof for treating hemophilia a |
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| AU7676894A (en) | 1993-08-27 | 1995-03-21 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Convection-enhanced drug delivery |
| AT401270B (de) | 1994-09-26 | 1996-07-25 | Immuno Ag | Verfahren zur quantifizierung von genomischer dna |
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| US6458563B1 (en) * | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
| AU722624B2 (en) | 1996-09-06 | 2000-08-10 | Trustees Of The University Of Pennsylvania, The | An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase |
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| WO2002024723A1 (en) * | 2000-09-19 | 2002-03-28 | Emory University | Modified factor viii |
| DE102004037611B4 (de) * | 2004-08-03 | 2013-10-02 | Geneart Ag | Induzierbare Genexpression |
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| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
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| EP2748185A1 (en) | 2011-08-24 | 2014-07-02 | The Board of Trustees of The Leland Stanford Junior University | New aav capsid proteins for nucleic acid transfer |
| BR112014019901A8 (pt) * | 2012-02-15 | 2018-01-02 | Biogen Idec Inc | Proteínas de fator viii recombinante |
| DK2839014T3 (da) | 2012-04-18 | 2021-03-08 | Childrens Hospital Philadelphia | Sammensætning og fremgangsmåder til højeffektiv genoverførsel ved anvendelse af aav-capsid-varianter |
| US20140271550A1 (en) | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
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| CN108473976B (zh) | 2015-10-28 | 2022-07-19 | 桑格摩生物治疗股份有限公司 | 肝特异性构建体、因子viii表达盒及其使用方法 |
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