RU2018139201A - Улучшенные способы доставки генов - Google Patents
Улучшенные способы доставки генов Download PDFInfo
- Publication number
- RU2018139201A RU2018139201A RU2018139201A RU2018139201A RU2018139201A RU 2018139201 A RU2018139201 A RU 2018139201A RU 2018139201 A RU2018139201 A RU 2018139201A RU 2018139201 A RU2018139201 A RU 2018139201A RU 2018139201 A RU2018139201 A RU 2018139201A
- Authority
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- Russia
- Prior art keywords
- endothelial cells
- mediated
- transduction
- nucleic acid
- carried out
- Prior art date
Links
- 238000000034 method Methods 0.000 title claims 17
- 238000001476 gene delivery Methods 0.000 title 1
- 210000002889 endothelial cell Anatomy 0.000 claims 8
- 230000001404 mediated effect Effects 0.000 claims 8
- 238000010361 transduction Methods 0.000 claims 7
- 230000026683 transduction Effects 0.000 claims 7
- 150000007523 nucleic acids Chemical class 0.000 claims 6
- 102000039446 nucleic acids Human genes 0.000 claims 6
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 238000001890 transfection Methods 0.000 claims 5
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 3
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims 3
- 210000004027 cell Anatomy 0.000 claims 3
- 206010021143 Hypoxia Diseases 0.000 claims 2
- 238000003501 co-culture Methods 0.000 claims 2
- 238000012239 gene modification Methods 0.000 claims 2
- 230000005017 genetic modification Effects 0.000 claims 2
- 235000013617 genetically modified food Nutrition 0.000 claims 2
- 230000001146 hypoxic effect Effects 0.000 claims 2
- 210000003606 umbilical vein Anatomy 0.000 claims 2
- 229920002307 Dextran Polymers 0.000 claims 1
- 229920000209 Hexadimethrine bromide Polymers 0.000 claims 1
- 101001105486 Homo sapiens Proteasome subunit alpha type-7 Proteins 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 102100021201 Proteasome subunit alpha type-7 Human genes 0.000 claims 1
- 241000700584 Simplexvirus Species 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical compound [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims 1
- 210000001185 bone marrow Anatomy 0.000 claims 1
- 229910000389 calcium phosphate Inorganic materials 0.000 claims 1
- 239000001506 calcium phosphate Substances 0.000 claims 1
- 235000011010 calcium phosphates Nutrition 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 238000004520 electroporation Methods 0.000 claims 1
- 210000004700 fetal blood Anatomy 0.000 claims 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 238000000520 microinjection Methods 0.000 claims 1
- 239000011859 microparticle Substances 0.000 claims 1
- 229910052760 oxygen Inorganic materials 0.000 claims 1
- 239000001301 oxygen Substances 0.000 claims 1
- 210000005259 peripheral blood Anatomy 0.000 claims 1
- 239000011886 peripheral blood Substances 0.000 claims 1
- 239000013600 plasmid vector Substances 0.000 claims 1
- 238000001556 precipitation Methods 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241001430294 unidentified retrovirus Species 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 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
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
-
- 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
-
- 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
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
-
- 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
- A61K35/44—Vessels; Vascular smooth muscle cells; Endothelial cells; Endothelial progenitor cells
-
- 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
- A61K35/48—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
-
- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/10—Cells modified by introduction of foreign genetic material
-
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Cell Biology (AREA)
- Zoology (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Developmental Biology & Embryology (AREA)
- General Health & Medical Sciences (AREA)
- Immunology (AREA)
- Virology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Reproductive Health (AREA)
- Hematology (AREA)
- Vascular Medicine (AREA)
- Gynecology & Obstetrics (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Claims (19)
1. Улучшенный способ генетического модифицирования CD34+ гемопоэтических стволовых или прогениторных клеток (HSPC), причем способ предусматривает
(a) совместное культивирование CD34+ HSPC-клеток с E4ORF1+ эндотелиальными клетками и
(b) приведение HSPC в контакт с одной или несколькими экзогенными молекулами нуклеиновой кислоты,
где стадию совместного культивирования начинают либо: до приведения HSPC в контакт с одной или несколькими экзогенными молекулами нуклеиновой кислоты, одновременно с ним или после него,
в результате чего обеспечивается генетическое модифицирование CD34+ HSPC.
2. Способ по п. 1, в котором стадию совместного культивирования осуществляют в гипоксических условиях.
3. Способ по п. 1, в котором стадию совместного культивирования осуществляют в сильно гипоксических условиях.
4. Способ по п. 1, в котором стадию совместного культивирования осуществляют при уровнях кислорода, находящихся в диапазоне от 0,1% до 18%.
5. Способ по п. 1, в котором HSPC происходят из костного мозга, периферической крови или пуповинной крови.
6. Способ по п. 1, в котором эндотелиальные клетки представляют собой эндотелиальные клетки человека.
7. Способ по п. 1, в котором эндотелиальные клетки представляют собой эндотелиальные клетки пупочной вены.
8. Способ по п. 1, в котором эндотелиальные клетки представляют собой эндотелиальные клетки пупочной вены человека.
9. Способ по п. 1, в котором эндотелиальные клетки являются митотически инактивированными.
10. Способ по п. 1, в котором стадию приведения целевых клеток в контакт с одной или несколькими экзогенными молекулами нуклеиновой кислоты осуществляют с помощью трансфекции.
11. Способ по п. 10, в котором трансфекция предусматривает опосредованную липосомами трансфекцию, опосредованную полибреном трансфекцию, опосредованную DEAE-декстраном трансфекцию, электропорацию, осаждение фосфатом кальция, микроинъекцию или бомбардировку микрочастицами.
12. Способ по п. 1, в котором стадию приведения целевых клеток в контакт с одной или несколькими экзогенными молекулами нуклеиновой кислоты осуществляют с помощью трансдукции.
13. Способ по п. 12, в котором трансдукцию осуществляют с применением опосредованной лентивирусом трансдукции, опосредованной аденовирусом трансдукции, опосредованной ретровирусом трансдукции, опосредованной аденоассоциированным вирусом трансдукции или опосредованной вирусом герпеса трансдукции.
14. Способ по п. 1, в котором молекула нуклеиновой кислоты присутствует в плазмидном векторе.
15. Способ по п. 1, в котором молекула нуклеиновой кислоты присутствует в вирусном векторе.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662323476P | 2016-04-15 | 2016-04-15 | |
| US62/323,476 | 2016-04-15 | ||
| US201662403110P | 2016-10-01 | 2016-10-01 | |
| US62/403,110 | 2016-10-01 | ||
| PCT/US2017/027884 WO2017181168A1 (en) | 2016-04-15 | 2017-04-17 | Enhanced gene delivery methods |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| RU2018139201A true RU2018139201A (ru) | 2020-05-15 |
| RU2018139201A3 RU2018139201A3 (ru) | 2020-11-13 |
Family
ID=60041985
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2018139201A RU2018139201A (ru) | 2016-04-15 | 2017-04-17 | Улучшенные способы доставки генов |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20190127760A1 (ru) |
| EP (1) | EP3442544B1 (ru) |
| JP (1) | JP7080184B2 (ru) |
| KR (1) | KR102428606B1 (ru) |
| CN (1) | CN109475582A (ru) |
| AU (1) | AU2017248848B2 (ru) |
| CA (1) | CA3021090A1 (ru) |
| IL (1) | IL262310B2 (ru) |
| RU (1) | RU2018139201A (ru) |
| SG (1) | SG11201809049UA (ru) |
| WO (1) | WO2017181168A1 (ru) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL265352B2 (en) | 2016-09-13 | 2024-05-01 | Angiocrine Bioscience Inc | Blood brain barrier containing engineered endothelial cells |
| CN110075319A (zh) * | 2019-04-29 | 2019-08-02 | 白涛 | 一种抗遗传病生物导弹 |
| US20230256029A1 (en) * | 2020-06-25 | 2023-08-17 | Angiocrine Bioscience, Inc. | Endothelial cells for mitigation of chemotherapy-induced toxicity |
| WO2023081277A1 (en) * | 2021-11-03 | 2023-05-11 | Memorial Sloan-Kettering Cancer Center | Epigenetic targets in clonal hematopoiesis |
| WO2024143555A1 (ja) * | 2022-12-28 | 2024-07-04 | 国立大学法人千葉大学 | 細胞分化度の調節方法 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003014313A2 (en) * | 2001-08-06 | 2003-02-20 | Bresagen, Ltd. | Alternative compositions and methods for the culture of stem cells |
| WO2006030442A2 (en) * | 2004-09-16 | 2006-03-23 | Gamida-Cell Ltd. | Methods of ex vivo progenitor and stem cell expansion by co-culture with mesenchymal cells |
| US20080173788A1 (en) * | 2007-01-18 | 2008-07-24 | Western Forms, Inc. | Lightweight Crane-Set Forming Panel |
| WO2008089448A2 (en) | 2007-01-19 | 2008-07-24 | Cornell Presearch Foundation, Inc. | Methods and compositions for promoting survival & proliferation of endothelial cells & stimulating angiogenesis |
| WO2014113415A1 (en) * | 2013-01-15 | 2014-07-24 | Cornell University | Reprogramming of human endothelium into hematopoietic multi-lineage progenitors by defined factors |
| US9790393B2 (en) | 2013-03-13 | 2017-10-17 | Cabot Corporation | Coatings having filler-polymer compositions with combined low dielectric constant, high resistivity, and optical density properties and controlled electrical resistivity, devices made therewith, and methods for making same |
| PL2970912T3 (pl) * | 2013-03-13 | 2019-08-30 | Wisconsin Alumni Research Foundation | Sposoby i materiały do hematopoetyczno-śródbłonkowego różnicowania ludzkich pluripotentnych komórek macierzystych w zdefiniowanych warunkach |
| EP3057432B1 (en) * | 2013-10-17 | 2018-11-21 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
-
2017
- 2017-04-17 EP EP17783351.4A patent/EP3442544B1/en active Active
- 2017-04-17 SG SG11201809049UA patent/SG11201809049UA/en unknown
- 2017-04-17 US US16/092,870 patent/US20190127760A1/en not_active Abandoned
- 2017-04-17 KR KR1020187032610A patent/KR102428606B1/ko active IP Right Grant
- 2017-04-17 JP JP2018554116A patent/JP7080184B2/ja active Active
- 2017-04-17 AU AU2017248848A patent/AU2017248848B2/en active Active
- 2017-04-17 WO PCT/US2017/027884 patent/WO2017181168A1/en active Application Filing
- 2017-04-17 RU RU2018139201A patent/RU2018139201A/ru unknown
- 2017-04-17 IL IL262310A patent/IL262310B2/en unknown
- 2017-04-17 CA CA3021090A patent/CA3021090A1/en active Pending
- 2017-04-17 CN CN201780036403.3A patent/CN109475582A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20190127760A1 (en) | 2019-05-02 |
| AU2017248848B2 (en) | 2022-12-08 |
| AU2017248848A1 (en) | 2018-11-22 |
| KR102428606B1 (ko) | 2022-08-02 |
| IL262310B1 (en) | 2024-02-01 |
| CN109475582A (zh) | 2019-03-15 |
| EP3442544A1 (en) | 2019-02-20 |
| EP3442544B1 (en) | 2024-05-29 |
| EP3442544C0 (en) | 2024-05-29 |
| SG11201809049UA (en) | 2018-11-29 |
| RU2018139201A3 (ru) | 2020-11-13 |
| EP3442544A4 (en) | 2020-01-01 |
| IL262310A (en) | 2018-11-29 |
| JP2019511238A (ja) | 2019-04-25 |
| JP7080184B2 (ja) | 2022-06-03 |
| WO2017181168A1 (en) | 2017-10-19 |
| IL262310B2 (en) | 2024-06-01 |
| KR20180133484A (ko) | 2018-12-14 |
| CA3021090A1 (en) | 2017-10-19 |
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