MX2020004092A - Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas. - Google Patents
Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas.Info
- Publication number
- MX2020004092A MX2020004092A MX2020004092A MX2020004092A MX2020004092A MX 2020004092 A MX2020004092 A MX 2020004092A MX 2020004092 A MX2020004092 A MX 2020004092A MX 2020004092 A MX2020004092 A MX 2020004092A MX 2020004092 A MX2020004092 A MX 2020004092A
- Authority
- MX
- Mexico
- Prior art keywords
- adeno
- associated virus
- methods
- restoring
- gene function
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 3
- 101150013707 HBB gene Proteins 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 102100021519 Hemoglobin subunit beta Human genes 0.000 abstract 2
- 206010064571 Gene mutation Diseases 0.000 abstract 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
- 238000004806 packaging method and process Methods 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
-
- 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
-
- 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
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
-
- 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
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61P7/06—Antianaemics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
- C07K14/01—DNA viruses
- C07K14/075—Adenoviridae
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/795—Porphyrin- or corrin-ring-containing peptides
- C07K14/805—Haemoglobins; Myoglobins
-
- 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
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- 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
- A01K2207/00—Modified animals
- A01K2207/12—Animals modified by administration of exogenous cells
-
- 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
- A01K2267/0381—Animal model for diseases of the hematopoietic 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
- 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)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Developmental Biology & Embryology (AREA)
- Immunology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762574163P | 2017-10-18 | 2017-10-18 | |
| US201862621102P | 2018-01-24 | 2018-01-24 | |
| PCT/US2018/056271 WO2019079437A1 (en) | 2017-10-18 | 2018-10-17 | ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING THE FUNCTION OF THE HBB GENE AND METHODS OF USE |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2020004092A true MX2020004092A (es) | 2021-01-08 |
Family
ID=64332161
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020004092A MX2020004092A (es) | 2017-10-18 | 2018-10-17 | Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US12201657B2 (enExample) |
| EP (1) | EP3697450A1 (enExample) |
| JP (1) | JP2021500070A (enExample) |
| KR (1) | KR20200095462A (enExample) |
| CN (1) | CN111447954A (enExample) |
| AU (1) | AU2018352855B2 (enExample) |
| BR (1) | BR112020007765A2 (enExample) |
| CA (1) | CA3079437A1 (enExample) |
| IL (1) | IL273877A (enExample) |
| MX (1) | MX2020004092A (enExample) |
| SG (1) | SG11202002737PA (enExample) |
| WO (1) | WO2019079437A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109536494A (zh) * | 2017-10-31 | 2019-03-29 | 广东赤萌医疗科技有限公司 | 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒 |
| CA3166374A1 (en) * | 2019-12-31 | 2021-07-08 | Swanbio Therapeutics Limited | Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn) |
| CN111363756B (zh) * | 2020-04-02 | 2023-01-17 | 中国医学科学院血液病医院(中国医学科学院血液学研究所) | 一种珠蛋白基因双表达慢病毒载体及其应用 |
| BR112022019182A2 (pt) * | 2020-04-06 | 2022-11-29 | Homology Medicines Inc | Composições de vírus adeno-associadas para transferência do gene ids e métodos de uso da mesma |
| EP4323513A4 (en) * | 2021-04-12 | 2025-09-17 | Kamau Therapeutics Inc | METHODS AND COMPOSITIONS FOR THE PRODUCTION OF GENETICALLY MODIFIED PRIMARY CELLS |
| WO2023019185A1 (en) * | 2021-08-10 | 2023-02-16 | Gentibio, Inc. | Compositions and methods for engineering stable tregs |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5449759A (en) * | 1987-05-16 | 1995-09-12 | Somatogen, Inc. | Hemoglobins with intersubunit desulfide bonds |
| US5252479A (en) * | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| EP0938578B1 (en) * | 1996-12-05 | 2004-02-04 | Crucell Holland B.V. | Genetic modification of primate hemopoietic repopulating stem cells |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| CN101348786B (zh) * | 2007-07-20 | 2011-05-11 | 谭孟群 | 一种人β珠蛋白基因及其重组腺相关病毒载体 |
| WO2009013559A1 (en) * | 2007-07-23 | 2009-01-29 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof |
| US8628966B2 (en) * | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| US11242525B2 (en) * | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| ES2746529T3 (es) | 2014-09-04 | 2020-03-06 | Memorial Sloan Kettering Cancer Center | Terapia génica de globina para tratar hemoglobinopatías |
| PL3198018T3 (pl) | 2014-09-24 | 2021-07-19 | City Of Hope | Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby |
| US20180135074A1 (en) * | 2015-02-19 | 2018-05-17 | University Of Florida Research Foundation, Incorporated | Recombinant aav vectors for gene therapy of human hematopoietic disorders |
| AU2017221405A1 (en) * | 2016-02-16 | 2018-09-20 | Carnegie Mellon University | Compositions for enhancing targeted gene editing and methods of use thereof |
-
2018
- 2018-10-17 WO PCT/US2018/056271 patent/WO2019079437A1/en not_active Ceased
- 2018-10-17 CN CN201880079840.8A patent/CN111447954A/zh active Pending
- 2018-10-17 KR KR1020207012240A patent/KR20200095462A/ko not_active Ceased
- 2018-10-17 BR BR112020007765-8A patent/BR112020007765A2/pt unknown
- 2018-10-17 EP EP18804159.4A patent/EP3697450A1/en not_active Withdrawn
- 2018-10-17 JP JP2020542536A patent/JP2021500070A/ja active Pending
- 2018-10-17 US US16/163,061 patent/US12201657B2/en active Active
- 2018-10-17 SG SG11202002737PA patent/SG11202002737PA/en unknown
- 2018-10-17 CA CA3079437A patent/CA3079437A1/en active Pending
- 2018-10-17 AU AU2018352855A patent/AU2018352855B2/en active Active
- 2018-10-17 MX MX2020004092A patent/MX2020004092A/es unknown
-
2020
- 2020-04-07 IL IL273877A patent/IL273877A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20190134118A1 (en) | 2019-05-09 |
| CA3079437A1 (en) | 2019-04-25 |
| EP3697450A1 (en) | 2020-08-26 |
| AU2018352855B2 (en) | 2022-07-14 |
| IL273877A (en) | 2020-05-31 |
| WO2019079437A1 (en) | 2019-04-25 |
| US12201657B2 (en) | 2025-01-21 |
| CN111447954A (zh) | 2020-07-24 |
| KR20200095462A (ko) | 2020-08-10 |
| AU2018352855A1 (en) | 2020-04-02 |
| SG11202002737PA (en) | 2020-05-28 |
| BR112020007765A2 (pt) | 2020-10-20 |
| JP2021500070A (ja) | 2021-01-07 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| MX2020004092A (es) | Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas. | |
| MX2017012097A (es) | Variantes de virus adenoasociado y métodos de uso de estas. | |
| BR112016018598A2 (pt) | vetor de vírus adeno-associados | |
| CO2020010783A2 (es) | Composiciones de virus adeno-asociadas para la transferencia de genes pah y métodos de uso de los mismos | |
| CO2019011450A2 (es) | Expresión transgénica selectiva de tejidos | |
| CO2018000134A2 (es) | Factor ix modificado, y composiciones para la transferencia de genes a células, órganos y tejidos | |
| PE20190563A1 (es) | Genes de mini-distrofina optimizados y casetes de expresion y su uso | |
| MX2022006652A (es) | Proteinas de capside modificadas para un suministro mejorado de vectores de parvovirus. | |
| CL2017002731A1 (es) | Adenovirus oncolítico que codifica una proteína b7 | |
| CO2017011344A2 (es) | Producción de vectores adeno-asociados de gran tamaño | |
| CO2021016797A2 (es) | Composiciones de virus adenoasociadas para la transferencia del gen arsa y métodos de uso de las mismas | |
| AU2016262093A8 (en) | Enhancing endonuclease based gene editing in primary cells | |
| MX2020005187A (es) | Compuestos y composiciones farmaceuticas antivirales, asi como metodos de uso de los mismos. | |
| PE20150163A1 (es) | Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav | |
| WO2015187825A3 (en) | Compositions and methods for modulating dysferlin expression | |
| MX2020000676A (es) | Composiciones y metodos para tratar beta-hemoglobinopatias. | |
| CO2020010793A2 (es) | Composiciones de virus adeno-asociado para restaurar la función del gen pah y métodos de uso de las mismas | |
| AR108017A1 (es) | Restablecimiento de la fertilidad masculina en el trigo | |
| PE20230433A1 (es) | Composiciones de virus adenoasociados para restaurar la funcion del gen pah y los metodos de uso de estas | |
| CO2021007958A2 (es) | Virus del herpes aviar recombinantes que contienen múltiples genes extraños | |
| EA201791233A1 (ru) | Материал стволовых клеток и способ его получения | |
| Schwieger et al. | Part I, Free actions of compact Abelian groups on C⁎-algebras | |
| WO2016069518A3 (en) | Genetically stable replication competent sendai virus vector(s) containing and expressing optimized hiv genes | |
| Tacchella | On a family of quivers related to the Gibbons–Hermsen system | |
| Gabriyelyan | Bounded subgroups as a von Neumann radical of an Abelian group |