RU2019120663A - Генная терапия для лечения мукополисахаридоза ii типа - Google Patents
Генная терапия для лечения мукополисахаридоза ii типа Download PDFInfo
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- RU2019120663A RU2019120663A RU2019120663A RU2019120663A RU2019120663A RU 2019120663 A RU2019120663 A RU 2019120663A RU 2019120663 A RU2019120663 A RU 2019120663A RU 2019120663 A RU2019120663 A RU 2019120663A RU 2019120663 A RU2019120663 A RU 2019120663A
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- promoter
- polynucleotide
- ltr
- polynucleotide according
- hiv
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- 201000002273 mucopolysaccharidosis II Diseases 0.000 title claims 5
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 title claims 5
- 238000001415 gene therapy Methods 0.000 title 1
- 108091033319 polynucleotide Proteins 0.000 claims 38
- 102000040430 polynucleotide Human genes 0.000 claims 38
- 239000002157 polynucleotide Substances 0.000 claims 38
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims 10
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 claims 10
- 229920001184 polypeptide Polymers 0.000 claims 9
- 108090000765 processed proteins & peptides Proteins 0.000 claims 9
- 102000004196 processed proteins & peptides Human genes 0.000 claims 9
- 210000004027 cell Anatomy 0.000 claims 8
- 210000004962 mammalian cell Anatomy 0.000 claims 8
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims 6
- 241000700605 Viruses Species 0.000 claims 5
- 238000000034 method Methods 0.000 claims 5
- 241000701022 Cytomegalovirus Species 0.000 claims 3
- 241000725303 Human immunodeficiency virus Species 0.000 claims 3
- 241000713666 Lentivirus Species 0.000 claims 3
- 238000012217 deletion Methods 0.000 claims 3
- 230000037430 deletion Effects 0.000 claims 3
- 238000012856 packing Methods 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 230000001177 retroviral effect Effects 0.000 claims 3
- 208000024891 symptom Diseases 0.000 claims 3
- 108091010877 Allograft inflammatory factor 1 Proteins 0.000 claims 2
- 102100040121 Allograft inflammatory factor 1 Human genes 0.000 claims 2
- 241000713704 Bovine immunodeficiency virus Species 0.000 claims 2
- 241000282693 Cercopithecidae Species 0.000 claims 2
- 241000713800 Feline immunodeficiency virus Species 0.000 claims 2
- 101000740205 Homo sapiens Sal-like protein 1 Proteins 0.000 claims 2
- 241000713340 Human immunodeficiency virus 2 Species 0.000 claims 2
- 102100022338 Integrin alpha-M Human genes 0.000 claims 2
- 102100037204 Sal-like protein 1 Human genes 0.000 claims 2
- 102100037029 Transmembrane protein 119 Human genes 0.000 claims 2
- 101710170979 Transmembrane protein 119 Proteins 0.000 claims 2
- 230000003247 decreasing effect Effects 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 210000000130 stem cell Anatomy 0.000 claims 2
- 108010017009 CD11b Antigen Proteins 0.000 claims 1
- 108090000835 CX3C Chemokine Receptor 1 Proteins 0.000 claims 1
- 102100039196 CX3C chemokine receptor 1 Human genes 0.000 claims 1
- 241000283707 Capra Species 0.000 claims 1
- 102000009410 Chemokine receptor Human genes 0.000 claims 1
- 108050000299 Chemokine receptor Proteins 0.000 claims 1
- 208000000059 Dyspnea Diseases 0.000 claims 1
- 206010013975 Dyspnoeas Diseases 0.000 claims 1
- 241000713730 Equine infectious anemia virus Species 0.000 claims 1
- 108091006027 G proteins Proteins 0.000 claims 1
- 102000030782 GTP binding Human genes 0.000 claims 1
- 108091000058 GTP-Binding Proteins 0.000 claims 1
- 208000031886 HIV Infections Diseases 0.000 claims 1
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 1
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims 1
- 101001046686 Homo sapiens Integrin alpha-M Proteins 0.000 claims 1
- 101000934372 Homo sapiens Macrosialin Proteins 0.000 claims 1
- 206010061598 Immunodeficiency Diseases 0.000 claims 1
- 208000029462 Immunodeficiency disease Diseases 0.000 claims 1
- 206010023230 Joint stiffness Diseases 0.000 claims 1
- 102100025136 Macrosialin Human genes 0.000 claims 1
- 108010057466 NF-kappa B Proteins 0.000 claims 1
- 102000003945 NF-kappa B Human genes 0.000 claims 1
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 claims 1
- 108010077524 Peptide Elongation Factor 1 Proteins 0.000 claims 1
- 241000714474 Rous sarcoma virus Species 0.000 claims 1
- 206010039491 Sarcoma Diseases 0.000 claims 1
- 108700026226 TATA Box Proteins 0.000 claims 1
- 108091023040 Transcription factor Proteins 0.000 claims 1
- 102000040945 Transcription factor Human genes 0.000 claims 1
- 208000010094 Visna Diseases 0.000 claims 1
- 238000009825 accumulation Methods 0.000 claims 1
- 102000019997 adhesion receptor Human genes 0.000 claims 1
- 108010013985 adhesion receptor Proteins 0.000 claims 1
- 206010003246 arthritis Diseases 0.000 claims 1
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 230000003920 cognitive function Effects 0.000 claims 1
- 239000002299 complementary DNA Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 206010014599 encephalitis Diseases 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 1
- 230000007813 immunodeficiency Effects 0.000 claims 1
- 102000006495 integrins Human genes 0.000 claims 1
- 108010044426 integrins Proteins 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 230000007659 motor function Effects 0.000 claims 1
- 239000013642 negative control Substances 0.000 claims 1
- 210000000056 organ Anatomy 0.000 claims 1
- 208000013220 shortness of breath Diseases 0.000 claims 1
- 230000009747 swallowing Effects 0.000 claims 1
- 230000008719 thickening Effects 0.000 claims 1
- 230000029812 viral genome replication Effects 0.000 claims 1
- 230000006490 viral transcription Effects 0.000 claims 1
Classifications
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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
-
- 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
- A61K35/33—Fibroblasts
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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
- A61K35/48—Reproductive organs
- A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
- A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
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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
-
- 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
- A61K48/0083—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 administration regime
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
-
- 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
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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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- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
- C12N5/0656—Adult fibroblasts
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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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
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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
- C12N2510/00—Genetically modified cells
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15034—Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—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
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- General Engineering & Computer Science (AREA)
- Cell Biology (AREA)
- Biochemistry (AREA)
- Developmental Biology & Embryology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Reproductive Health (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Gynecology & Obstetrics (AREA)
- Diabetes (AREA)
- Rheumatology (AREA)
- Obesity (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662430819P | 2016-12-06 | 2016-12-06 | |
| US62/430,819 | 2016-12-06 | ||
| PCT/US2017/064940 WO2018106821A1 (en) | 2016-12-06 | 2017-12-06 | Gene therapy for mucopolysaccharidosis, type ii |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2019120663A true RU2019120663A (ru) | 2021-01-11 |
Family
ID=62492351
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2019120663A RU2019120663A (ru) | 2016-12-06 | 2017-12-06 | Генная терапия для лечения мукополисахаридоза ii типа |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20200071721A1 (enExample) |
| EP (1) | EP3562937A4 (enExample) |
| JP (1) | JP2020500562A (enExample) |
| KR (1) | KR20190088554A (enExample) |
| CN (1) | CN110199028A (enExample) |
| AU (1) | AU2017370673A1 (enExample) |
| BR (1) | BR112019011590A2 (enExample) |
| CA (1) | CA3046080A1 (enExample) |
| IL (1) | IL267060A (enExample) |
| MA (1) | MA47173A (enExample) |
| MX (1) | MX2019006551A (enExample) |
| RU (1) | RU2019120663A (enExample) |
| WO (1) | WO2018106821A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109641063B (zh) | 2016-04-20 | 2022-11-15 | 能源环境和技术研究中心O.A., M.P. | 用于增强pklr的基因表达的组合物和方法 |
| 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 |
| JP7576754B2 (ja) | 2018-04-11 | 2024-11-01 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | 幹細胞移植のための組成物および方法 |
| MX2021001292A (es) * | 2018-07-30 | 2021-07-15 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Metodos para la modificacion genica de celulas hematopoyeticas. |
| WO2022212616A1 (en) * | 2021-04-01 | 2022-10-06 | The University Of North Carolina At Chapel Hill | Aav-ids vectors for treatment of mucopolysaccharidosis ii |
| WO2024233287A1 (en) * | 2023-05-05 | 2024-11-14 | The Regents Of The University Of California | Viral vectors for cell therapy |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2872170B1 (fr) * | 2004-06-25 | 2006-11-10 | Centre Nat Rech Scient Cnrse | Lentivirus non interactif et non replicatif, preparation et utilisations |
| US20110294114A1 (en) * | 2009-12-04 | 2011-12-01 | Cincinnati Children's Hospital Medical Center | Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells |
| AU2012267512B2 (en) * | 2011-06-10 | 2014-11-20 | Bluebird Bio, Inc. | Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy |
| US9695443B2 (en) * | 2012-05-25 | 2017-07-04 | Commissariat A L'energie Atomique Et Aux Energies Alternatives | Vector for the selective silencing of a gene in astrocytes |
| WO2014201252A2 (en) * | 2013-06-13 | 2014-12-18 | Shire Human Genetic Therapies, Inc. | Messenger rna based viral production |
-
2017
- 2017-12-06 AU AU2017370673A patent/AU2017370673A1/en not_active Abandoned
- 2017-12-06 US US16/466,123 patent/US20200071721A1/en not_active Abandoned
- 2017-12-06 BR BR112019011590A patent/BR112019011590A2/pt not_active IP Right Cessation
- 2017-12-06 JP JP2019551257A patent/JP2020500562A/ja active Pending
- 2017-12-06 RU RU2019120663A patent/RU2019120663A/ru not_active Application Discontinuation
- 2017-12-06 CN CN201780083277.7A patent/CN110199028A/zh active Pending
- 2017-12-06 WO PCT/US2017/064940 patent/WO2018106821A1/en not_active Ceased
- 2017-12-06 CA CA3046080A patent/CA3046080A1/en not_active Abandoned
- 2017-12-06 MX MX2019006551A patent/MX2019006551A/es unknown
- 2017-12-06 EP EP17878786.7A patent/EP3562937A4/en not_active Withdrawn
- 2017-12-06 MA MA047173A patent/MA47173A/fr unknown
- 2017-12-06 KR KR1020197019345A patent/KR20190088554A/ko not_active Ceased
-
2019
- 2019-06-03 IL IL267060A patent/IL267060A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2018106821A1 (en) | 2018-06-14 |
| US20200071721A1 (en) | 2020-03-05 |
| BR112019011590A2 (pt) | 2019-10-22 |
| JP2020500562A (ja) | 2020-01-16 |
| CN110199028A (zh) | 2019-09-03 |
| MX2019006551A (es) | 2019-10-15 |
| EP3562937A4 (en) | 2020-09-16 |
| CA3046080A1 (en) | 2018-06-14 |
| AU2017370673A1 (en) | 2019-06-27 |
| EP3562937A1 (en) | 2019-11-06 |
| MA47173A (fr) | 2019-11-06 |
| IL267060A (en) | 2019-08-29 |
| KR20190088554A (ko) | 2019-07-26 |
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