CN110770344A - shRNA表达框、携带其的多核苷酸序列及其应用 - Google Patents
shRNA表达框、携带其的多核苷酸序列及其应用 Download PDFInfo
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- CN110770344A CN110770344A CN201880034603.XA CN201880034603A CN110770344A CN 110770344 A CN110770344 A CN 110770344A CN 201880034603 A CN201880034603 A CN 201880034603A CN 110770344 A CN110770344 A CN 110770344A
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- sequence
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- expression cassette
- aav
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- 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
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- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
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- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
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- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
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- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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- 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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- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
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- 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
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- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- 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
- C12N2750/14171—Demonstrated in vivo effect
Abstract
Description
Claims (29)
- PCT国内申请,权利要求书已公开。
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CN201710210863 | 2017-03-31 | ||
CN2017102108635 | 2017-03-31 | ||
PCT/CN2018/080480 WO2018177244A1 (zh) | 2017-03-31 | 2018-03-26 | shRNA表达框、携带其的多核苷酸序列及其应用 |
Publications (1)
Publication Number | Publication Date |
---|---|
CN110770344A true CN110770344A (zh) | 2020-02-07 |
Family
ID=63674215
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CN201880034603.XA Pending CN110770344A (zh) | 2017-03-31 | 2018-03-26 | shRNA表达框、携带其的多核苷酸序列及其应用 |
Country Status (6)
Country | Link |
---|---|
US (1) | US20210108197A1 (zh) |
EP (1) | EP3613856A4 (zh) |
CN (1) | CN110770344A (zh) |
SG (1) | SG11201909136PA (zh) |
TW (1) | TW201837173A (zh) |
WO (1) | WO2018177244A1 (zh) |
Cited By (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN112107677A (zh) * | 2020-07-31 | 2020-12-22 | 江苏省人民医院(南京医科大学第一附属医院) | EFTUD2应用及Epro-LUC-HepG2建模法 |
WO2023125823A1 (zh) * | 2021-12-31 | 2023-07-06 | 北京三诺佳邑生物技术有限责任公司 | 靶向HIV的siRNA和shRNA及其相应的组合、表达盒、细胞及其应用 |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
BR112023009276A2 (pt) * | 2020-11-16 | 2024-02-06 | Beijing Solobio Genetechnology Co Ltd | Vírus recombinantes adenoassociados (raav), composição e uso do raav |
AU2022218196A1 (en) * | 2021-02-05 | 2023-08-10 | Maze Therapeutics, Inc. | Vectors comprising stuffer polynucleotide sequences |
Citations (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN1580070A (zh) * | 2003-08-06 | 2005-02-16 | 北京三诺佳邑生物技术有限责任公司 | 一组抗hbv感染及防治乙型肝炎的核苷酸序列及其应用 |
US20070027099A1 (en) * | 2003-05-19 | 2007-02-01 | Lin Marie C | Gene therapy of HBV infection via adeno-associated viral vector mediated long term expression of small hairpin RNA (shRNA) |
WO2014114934A1 (en) * | 2013-01-22 | 2014-07-31 | Horizon Discovery Limited | Methods for improving the efficiency of gene targeting |
CN105612253A (zh) * | 2013-03-15 | 2016-05-25 | 费城儿童医院 | 含有填充者/填充物多核苷酸序列的载体及其制备方法 |
Family Cites Families (10)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US8202979B2 (en) * | 2002-02-20 | 2012-06-19 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid |
ES2874298T3 (es) | 2003-09-30 | 2021-11-04 | Univ Pennsylvania | Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos |
WO2009137006A2 (en) | 2008-04-30 | 2009-11-12 | The University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
US9790502B2 (en) * | 2010-10-28 | 2017-10-17 | Benitec Biopharma Limited | HBV treatment |
CN103040450A (zh) * | 2011-10-14 | 2013-04-17 | 李玲 | 便捷式自身消毒血压计 |
CA2922500A1 (en) * | 2013-08-27 | 2015-03-05 | Research Institute At Nationwide Children's Hospital | Products and methods for treatment of amyotrophic lateral sclerosis |
MX2017010370A (es) * | 2015-02-10 | 2017-12-14 | Genzyme Corp | Suministro mejorado de particulas virales al cuerpo estriado y al cortex. |
CA3021949C (en) * | 2015-04-24 | 2023-10-17 | University Of Massachusetts | Modified aav constructs and uses thereof |
AU2017234929A1 (en) * | 2016-03-18 | 2018-10-04 | The Children's Hospital Of Philadelphia | Therapeutic for treatment of diseases including the central nervous system |
KR20190064590A (ko) * | 2016-09-14 | 2019-06-10 | 베니텍 바이오파마 리미티드 | 비-기능성 t-세포 수용체 (tcr)를 갖는 t-세포를 생산하기 위한 시약, 이를 포함하는 조성물 및 이의 용도 |
-
2018
- 2018-03-26 EP EP18777944.2A patent/EP3613856A4/en active Pending
- 2018-03-26 US US16/498,369 patent/US20210108197A1/en active Pending
- 2018-03-26 WO PCT/CN2018/080480 patent/WO2018177244A1/zh unknown
- 2018-03-26 CN CN201880034603.XA patent/CN110770344A/zh active Pending
- 2018-03-26 SG SG11201909136P patent/SG11201909136PA/en unknown
- 2018-03-27 TW TW107110446A patent/TW201837173A/zh unknown
Patent Citations (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20070027099A1 (en) * | 2003-05-19 | 2007-02-01 | Lin Marie C | Gene therapy of HBV infection via adeno-associated viral vector mediated long term expression of small hairpin RNA (shRNA) |
CN1580070A (zh) * | 2003-08-06 | 2005-02-16 | 北京三诺佳邑生物技术有限责任公司 | 一组抗hbv感染及防治乙型肝炎的核苷酸序列及其应用 |
WO2014114934A1 (en) * | 2013-01-22 | 2014-07-31 | Horizon Discovery Limited | Methods for improving the efficiency of gene targeting |
CN105612253A (zh) * | 2013-03-15 | 2016-05-25 | 费城儿童医院 | 含有填充者/填充物多核苷酸序列的载体及其制备方法 |
Non-Patent Citations (15)
Cited By (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN112107677A (zh) * | 2020-07-31 | 2020-12-22 | 江苏省人民医院(南京医科大学第一附属医院) | EFTUD2应用及Epro-LUC-HepG2建模法 |
WO2023125823A1 (zh) * | 2021-12-31 | 2023-07-06 | 北京三诺佳邑生物技术有限责任公司 | 靶向HIV的siRNA和shRNA及其相应的组合、表达盒、细胞及其应用 |
Also Published As
Publication number | Publication date |
---|---|
TW201837173A (zh) | 2018-10-16 |
EP3613856A4 (en) | 2020-12-23 |
SG11201909136PA (en) | 2019-10-30 |
US20210108197A1 (en) | 2021-04-15 |
WO2018177244A1 (zh) | 2018-10-04 |
EP3613856A1 (en) | 2020-02-26 |
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