MX2021012682A - Metodos y composiciones para la expresion del transgen. - Google Patents
Metodos y composiciones para la expresion del transgen.Info
- Publication number
- MX2021012682A MX2021012682A MX2021012682A MX2021012682A MX2021012682A MX 2021012682 A MX2021012682 A MX 2021012682A MX 2021012682 A MX2021012682 A MX 2021012682A MX 2021012682 A MX2021012682 A MX 2021012682A MX 2021012682 A MX2021012682 A MX 2021012682A
- Authority
- MX
- Mexico
- Prior art keywords
- transgene
- methods
- cell
- compositions
- transgene expression
- Prior art date
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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
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/69—Boron compounds
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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
- A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
- A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
- A61K31/704—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
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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
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/05—Dipeptides
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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
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/06—Tripeptides
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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
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/07—Tetrapeptides
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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
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
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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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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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
- C12N2750/14143—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)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Virology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Mycology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Marine Sciences & Fisheries (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Pulmonology (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)
- Apparatus Associated With Microorganisms And Enzymes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
La descripción provee métodos para la expresión de un transgén en una célula, métodos para el tratamiento de trastornos en un sujeto que lo necesita y composiciones farmacéuticas. En particular, los métodos implican poner una célula (por ejemplo, una célula de un sujeto que padece un trastorno como la fibrosis quística) en contacto con un virus adenoasociado recombinante (rAAV) que incluye, en una modalidad, una proteína de cápside AV.TL65 y un polinucleótido que incluye un transgén en combinación con un aumentador de la transducción de AAV, de forma que expresa el transgén en la célula. La descripción también provee composiciones farmacéuticas que incluyen un rAAV que incluye, en una modalidad, una proteína de cápside AV.TL65 y un polinucleótido que incluye un transgén en combinación con uno o más aumentadores.
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962833979P | 2019-04-15 | 2019-04-15 | |
| US201962926317P | 2019-10-25 | 2019-10-25 | |
| US202062967219P | 2020-01-29 | 2020-01-29 | |
| PCT/US2020/028269 WO2020214672A1 (en) | 2019-04-15 | 2020-04-15 | Methods and compositions for transgene expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021012682A true MX2021012682A (es) | 2022-03-25 |
Family
ID=70779846
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021012682A MX2021012682A (es) | 2019-04-15 | 2020-04-15 | Metodos y composiciones para la expresion del transgen. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220195461A1 (es) |
| EP (1) | EP3955970A1 (es) |
| JP (2) | JP2022529470A (es) |
| KR (1) | KR20220047538A (es) |
| CN (1) | CN114340683A (es) |
| AU (1) | AU2020257182A1 (es) |
| BR (1) | BR112021020706A2 (es) |
| CA (1) | CA3137078A1 (es) |
| IL (1) | IL287262A (es) |
| MX (1) | MX2021012682A (es) |
| SG (1) | SG11202111353QA (es) |
| WO (1) | WO2020214672A1 (es) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11702672B2 (en) | 2016-02-08 | 2023-07-18 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| US11684679B2 (en) | 2016-03-07 | 2023-06-27 | University Of Iowa Research Foundation | AAV-mediated expression using a synthetic promoter and enhancer |
| KR20220044899A (ko) * | 2019-04-15 | 2022-04-12 | 유니버시티 오브 아이오와 리써치 파운데이션 | 낭성 섬유증의 치료를 위한 조성물 및 방법 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| ES2216005T3 (es) | 1993-11-09 | 2004-10-16 | Targeted Genetics Corporation | Produccion de titulos elevados de vectores de aav recombinantes. |
| AU678867B2 (en) | 1993-11-09 | 1997-06-12 | Medical College Of Ohio, The | Stable cell lines capable of expressing the adeno-associated virus replication gene |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| US5656785A (en) | 1995-08-07 | 1997-08-12 | The Charles Stark Draper Laboratory, Inc. | Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor |
| WO1998023018A1 (en) | 1996-11-19 | 1998-05-28 | Surgx Corporation | A transient voltage protection device and method of making same |
| JP2001506133A (ja) | 1996-12-18 | 2001-05-15 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの産生における使用のための、ａａｖスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株 |
| CA2520028A1 (en) * | 2003-03-31 | 2004-10-21 | University Of Iowa Research Foundation | Compounds and methods to enhance raav transduction |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| WO2011097456A2 (en) * | 2010-02-05 | 2011-08-11 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| KR20210122917A (ko) * | 2013-03-14 | 2021-10-12 | 샤이어 휴먼 지네틱 테라피즈 인크. | Cftr mrna 조성물 및 관련 방법 및 사용 |
| JP6516725B2 (ja) * | 2013-04-08 | 2019-05-22 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | キメラアデノ随伴ウイルス／ボカウイルスパルボウイルスベクター |
| AU2015231439B2 (en) * | 2014-03-17 | 2019-11-14 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| US11702672B2 (en) * | 2016-02-08 | 2023-07-18 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| US11684679B2 (en) * | 2016-03-07 | 2023-06-27 | University Of Iowa Research Foundation | AAV-mediated expression using a synthetic promoter and enhancer |
| US11192925B2 (en) * | 2016-10-19 | 2021-12-07 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| CN106928336B (zh) * | 2017-01-20 | 2019-09-24 | 首都医科大学附属北京儿童医院 | 囊性纤维化患者的cftr基因突变形式及其应用 |
| CA3054687A1 (en) * | 2017-02-28 | 2018-09-07 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| CA3054711A1 (en) * | 2017-03-15 | 2018-09-20 | The University Of North Carolina At Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
| KR20220044899A (ko) * | 2019-04-15 | 2022-04-12 | 유니버시티 오브 아이오와 리써치 파운데이션 | 낭성 섬유증의 치료를 위한 조성물 및 방법 |
-
2020
- 2020-04-15 MX MX2021012682A patent/MX2021012682A/es unknown
- 2020-04-15 KR KR1020217037211A patent/KR20220047538A/ko not_active Application Discontinuation
- 2020-04-15 SG SG11202111353QA patent/SG11202111353QA/en unknown
- 2020-04-15 CN CN202080043579.3A patent/CN114340683A/zh active Pending
- 2020-04-15 US US17/603,840 patent/US20220195461A1/en active Pending
- 2020-04-15 EP EP20727413.5A patent/EP3955970A1/en active Pending
- 2020-04-15 AU AU2020257182A patent/AU2020257182A1/en active Pending
- 2020-04-15 BR BR112021020706A patent/BR112021020706A2/pt unknown
- 2020-04-15 JP JP2021561893A patent/JP2022529470A/ja active Pending
- 2020-04-15 CA CA3137078A patent/CA3137078A1/en active Pending
- 2020-04-15 WO PCT/US2020/028269 patent/WO2020214672A1/en unknown
-
2021
- 2021-10-14 IL IL287262A patent/IL287262A/en unknown
-
2023
- 2023-07-21 JP JP2023119196A patent/JP2023126658A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL287262A (en) | 2021-12-01 |
| BR112021020706A2 (pt) | 2022-03-15 |
| CA3137078A1 (en) | 2020-10-22 |
| JP2023126658A (ja) | 2023-09-07 |
| AU2020257182A1 (en) | 2021-12-09 |
| EP3955970A1 (en) | 2022-02-23 |
| CN114340683A (zh) | 2022-04-12 |
| US20220195461A1 (en) | 2022-06-23 |
| KR20220047538A (ko) | 2022-04-18 |
| SG11202111353QA (en) | 2021-11-29 |
| JP2022529470A (ja) | 2022-06-22 |
| WO2020214672A1 (en) | 2020-10-22 |
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