PE20231949A1 - VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA - Google Patents
VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIAInfo
- Publication number
- PE20231949A1 PE20231949A1 PE2023001716A PE2023001716A PE20231949A1 PE 20231949 A1 PE20231949 A1 PE 20231949A1 PE 2023001716 A PE2023001716 A PE 2023001716A PE 2023001716 A PE2023001716 A PE 2023001716A PE 20231949 A1 PE20231949 A1 PE 20231949A1
- Authority
- PE
- Peru
- Prior art keywords
- factor viii
- treatment
- seq
- acid variant
- hemostasy
- Prior art date
Links
- 229960000301 factor viii Drugs 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title 1
- 108010054218 Factor VIII Proteins 0.000 abstract 3
- 102000001690 Factor VIII Human genes 0.000 abstract 3
- 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
- 239000013607 AAV vector Substances 0.000 abstract 1
- 108090000565 Capsid Proteins Proteins 0.000 abstract 1
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 1
- 102100026735 Coagulation factor VIII Human genes 0.000 abstract 1
- 201000003542 Factor VIII deficiency Diseases 0.000 abstract 1
- 208000009292 Hemophilia A Diseases 0.000 abstract 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 abstract 1
- 239000002253 acid Substances 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 238000012217 deletion Methods 0.000 abstract 1
- 230000037430 deletion Effects 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 230000001747 exhibiting effect Effects 0.000 abstract 1
- 230000014509 gene expression Effects 0.000 abstract 1
- 239000002245 particle Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 238000013518 transcription Methods 0.000 abstract 1
- 230000035897 transcription Effects 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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- 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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- C12P21/00—Preparation of peptides or proteins
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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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- 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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- C12N2750/00011—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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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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- 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
- C12N2750/14145—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)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (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)
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Abstract
La presente invencion se refiere a una particula de AAV recombinante (rAAV) que comprende un genoma del vector AAV que comprende una variante de acido nucleico que codifica el Factor VIII (FVIII) que tiene una delecion del dominio B, en donde la variante de acido nucleico comprende la SEC ID NO: 1 a 18, entre otros, y en donde el rAAV comprende proteina de la capside de AAV VP1, tal como la capside LK03 de SEC ID NO: 27 o SPK de SEC ID NO: 28, VP2 y VP3. Tambien se refiere a una composicion farmaceutica y a una celula huesped. La variante de acido nucleico proporciona una transcripcion y expresion incrementadas, exhibiendo una actividad mejorada sobre proteinas de Factor VIII de tipo natural, siendo util en el tratamiento de la hemofilia A.
Applications Claiming Priority (5)
| 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 | |
| PCT/US2016/059793 WO2017075619A1 (en) | 2015-10-30 | 2016-10-31 | Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20231949A1 true PE20231949A1 (es) | 2023-12-05 |
Family
ID=58630834
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2023001716A PE20231949A1 (es) | 2015-10-30 | 2016-10-30 | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| PE2018000713A PE20181168A1 (es) | 2015-10-30 | 2016-10-31 | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2018000713A PE20181168A1 (es) | 2015-10-30 | 2016-10-31 | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA 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 |
| SG10202100131WA (en) | 2015-06-23 | 2021-02-25 | Childrens Hospital Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| 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 |
| KR102604510B1 (ko) | 2016-07-26 | 2023-11-23 | 바이오마린 파머수티컬 인크. | 신규 아데노-관련 바이러스 캡시드 단백질 |
| CA3071519A1 (en) * | 2017-08-01 | 2019-02-07 | Spark Therapeutics, Inc. | Factor viii (fviii) gene therapy methods |
| TWI904068B (zh) * | 2017-08-09 | 2025-11-11 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| KR101952102B1 (ko) * | 2017-12-07 | 2019-02-26 | 주식회사 지앤피바이오사이언스 | 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터 |
| US11739345B2 (en) | 2018-05-09 | 2023-08-29 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| MX2020012167A (es) | 2018-05-14 | 2022-07-13 | Biomarin Pharm Inc | Expresion estable de vectores de aav en sujetos juveniles. |
| 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 |
| CA3108799A1 (en) | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | 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 |
| CN113614236A (zh) * | 2018-11-16 | 2021-11-05 | 星火治疗有限公司 | 用于检测腺相关病毒(aav)载体转导的增强子和抑制剂和/或检测或定量抗aav结合抗体的体外测定 |
| GB201900702D0 (en) * | 2019-01-18 | 2019-03-06 | Univ Bristol | Therapy |
| SG11202108357PA (en) | 2019-02-15 | 2021-08-30 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| BR112021017853A2 (pt) * | 2019-03-13 | 2021-11-30 | Generation Bio Co | Vetores de dna não virais e usos dos mesmos para expressar terapêuticas de fviii |
| JP2023501262A (ja) * | 2019-11-01 | 2023-01-18 | フリーライン セラピューティクス リミテッド | 転写調節エレメント |
| TW202140793A (zh) * | 2020-01-16 | 2021-11-01 | 美商舒爾人類基因療法公司 | 用於苯酮尿症之基於腺相關病毒之基因療法 |
| 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 |
| PE20251838A1 (es) * | 2022-04-28 | 2025-07-17 | Biocad Joint Stock Co | Acido nucleico de codon optimizado que codifica la proteina del factor viii de coagulacion de dominio b suprimido, y uso del mismo |
| AU2023333051A1 (en) * | 2022-08-31 | 2025-03-20 | Hydrogene Therapeutics, Inc. | Methods and compositions for hydrodynamic gene delivery |
| 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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| Publication number | Priority date | Publication date | Assignee | Title |
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| US6093699A (en) | 1987-07-09 | 2000-07-25 | The University Of Manitoba | Method for gene therapy involving suppression of an immune response |
| WO1994017810A1 (en) | 1993-02-12 | 1994-08-18 | The Wistar Institute Of Anatomy And Biology | Recombinant cytomegalovirus vaccine |
| WO1994023744A1 (en) | 1993-04-16 | 1994-10-27 | The Wistar Institute Of Anatomy And Biology | Recombinant cytomegalovirus vaccine |
| WO1995005864A1 (en) | 1993-08-27 | 1995-03-02 | Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services | Convection-enhanced drug delivery |
| AT401270B (de) | 1994-09-26 | 1996-07-25 | Immuno Ag | Verfahren zur quantifizierung von genomischer dna |
| US5998205A (en) | 1994-11-28 | 1999-12-07 | Genetic Therapy, Inc. | Vectors for tissue-specific replication |
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