MX2021014337A - Vectores de genoterapia para osteopetrosis maligna infantil. - Google Patents
Vectores de genoterapia para osteopetrosis maligna infantil.Info
- Publication number
- MX2021014337A MX2021014337A MX2021014337A MX2021014337A MX2021014337A MX 2021014337 A MX2021014337 A MX 2021014337A MX 2021014337 A MX2021014337 A MX 2021014337A MX 2021014337 A MX2021014337 A MX 2021014337A MX 2021014337 A MX2021014337 A MX 2021014337A
- Authority
- MX
- Mexico
- Prior art keywords
- vectors
- genotherapy
- children
- malignant osteopetrosis
- disclosure provides
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 3
- 230000003211 malignant effect Effects 0.000 title 1
- 208000002865 osteopetrosis Diseases 0.000 title 1
- 208000035905 Autosomal recessive malignant osteopetrosis Diseases 0.000 abstract 2
- 101000854879 Homo sapiens V-type proton ATPase 116 kDa subunit a 2 Proteins 0.000 abstract 2
- 102100020745 V-type proton ATPase 116 kDa subunit a 2 Human genes 0.000 abstract 2
- 208000025213 autosomal recessive osteopetrosis Diseases 0.000 abstract 2
- 101000854875 Homo sapiens V-type proton ATPase 116 kDa subunit a 3 Proteins 0.000 abstract 1
- 102100020738 V-type proton ATPase 116 kDa subunit a 3 Human genes 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 229920001184 polypeptide Polymers 0.000 abstract 1
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 1
Classifications
-
- 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
-
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0387—Animal model for diseases of the immune system
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—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)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962852216P | 2019-05-23 | 2019-05-23 | |
| PCT/US2020/034394 WO2020237219A1 (en) | 2019-05-23 | 2020-05-22 | Gene therapy vectors for infantile malignant osteopetrosis |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021014337A true MX2021014337A (es) | 2022-03-17 |
Family
ID=73458718
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021014337A MX2021014337A (es) | 2019-05-23 | 2020-05-22 | Vectores de genoterapia para osteopetrosis maligna infantil. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220218844A1 (https=) |
| EP (1) | EP3973066A4 (https=) |
| JP (2) | JP7623299B2 (https=) |
| KR (1) | KR20220012266A (https=) |
| CN (1) | CN113994008A (https=) |
| AU (1) | AU2020278046A1 (https=) |
| BR (1) | BR112021023473A2 (https=) |
| CA (1) | CA3137700A1 (https=) |
| IL (1) | IL288193A (https=) |
| MX (1) | MX2021014337A (https=) |
| SG (1) | SG11202112347RA (https=) |
| WO (1) | WO2020237219A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HUE055862T2 (hu) | 2016-04-20 | 2021-12-28 | Centro De Investig Energeticas | Készítmények és eljárások PKLR fokozott génexpressziójára |
| SG11202002263TA (en) | 2017-10-16 | 2020-04-29 | Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas | Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency |
| WO2019200167A1 (en) | 2018-04-11 | 2019-10-17 | Rocket Pharmaceuticals, Ltd. | Compositions and methods for stem cell transplant |
| MX2020011386A (es) | 2018-04-27 | 2021-01-29 | Spacecraft Seven Llc | Terapia genica para la degeneracion del snc. |
| KR20230043869A (ko) | 2020-08-07 | 2023-03-31 | 스페이스크래프트 세븐, 엘엘씨 | Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법 |
| CN116042719B (zh) * | 2022-12-28 | 2025-11-11 | 蓝图生物医药(广州)有限公司 | 一种重组腺相关病毒载体及其应用 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| GB0202771D0 (en) * | 2002-02-06 | 2002-03-27 | Univ Aberdeen | Genetic markers for bone mass |
| CA2650507A1 (en) * | 2006-04-27 | 2007-11-08 | Universite De Montreal | Assessment and reduction of risk of graft-versus-host disease |
| AU2013309488A1 (en) * | 2012-08-29 | 2015-03-05 | Nature Technology Corporation | DNA plasmids with improved expression |
| WO2014077863A1 (en) * | 2012-11-19 | 2014-05-22 | Nature Technology Corporation | Replicative minicircle vectors with improved expression |
| MX374532B (es) * | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| CN116064678A (zh) * | 2016-01-15 | 2023-05-05 | 美国基因技术国际有限公司 | 用于活化γ-δT细胞的方法和组合物 |
| CN121102467A (zh) * | 2016-04-22 | 2025-12-12 | 克莱格医学有限公司 | 用于细胞免疫疗法的组合物和方法 |
| KR20240151726A (ko) | 2016-09-08 | 2024-10-18 | 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. | 판코니 빈혈 환자를 위한 유전자 치료 |
-
2020
- 2020-05-22 SG SG11202112347RA patent/SG11202112347RA/en unknown
- 2020-05-22 CA CA3137700A patent/CA3137700A1/en active Pending
- 2020-05-22 AU AU2020278046A patent/AU2020278046A1/en not_active Abandoned
- 2020-05-22 JP JP2021569298A patent/JP7623299B2/ja active Active
- 2020-05-22 BR BR112021023473A patent/BR112021023473A2/pt unknown
- 2020-05-22 US US17/612,134 patent/US20220218844A1/en not_active Abandoned
- 2020-05-22 CN CN202080044921.1A patent/CN113994008A/zh active Pending
- 2020-05-22 MX MX2021014337A patent/MX2021014337A/es unknown
- 2020-05-22 KR KR1020217040611A patent/KR20220012266A/ko not_active Ceased
- 2020-05-22 EP EP20809375.7A patent/EP3973066A4/en active Pending
- 2020-05-22 WO PCT/US2020/034394 patent/WO2020237219A1/en not_active Ceased
-
2021
- 2021-11-17 IL IL288193A patent/IL288193A/en unknown
-
2025
- 2025-01-16 JP JP2025005848A patent/JP2025063200A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3973066A1 (en) | 2022-03-30 |
| SG11202112347RA (en) | 2021-12-30 |
| EP3973066A4 (en) | 2023-07-26 |
| JP2025063200A (ja) | 2025-04-15 |
| WO2020237219A1 (en) | 2020-11-26 |
| KR20220012266A (ko) | 2022-02-03 |
| JP2022532802A (ja) | 2022-07-19 |
| CA3137700A1 (en) | 2020-11-26 |
| CN113994008A (zh) | 2022-01-28 |
| AU2020278046A1 (en) | 2022-01-27 |
| US20220218844A1 (en) | 2022-07-14 |
| JP7623299B2 (ja) | 2025-01-28 |
| IL288193A (en) | 2022-01-01 |
| BR112021023473A2 (pt) | 2022-02-01 |
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