CN112513271A - 使用营养缺陷型可调控细胞进行基因治疗的方法和组合物 - Google Patents
使用营养缺陷型可调控细胞进行基因治疗的方法和组合物 Download PDFInfo
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- CN112513271A CN112513271A CN201980031608.1A CN201980031608A CN112513271A CN 112513271 A CN112513271 A CN 112513271A CN 201980031608 A CN201980031608 A CN 201980031608A CN 112513271 A CN112513271 A CN 112513271A
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- 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
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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
- C12N15/1137—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 against enzymes
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- 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
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- C12N5/06—Animal cells or tissues; Human cells or tissues
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- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
- A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the route of administration
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- A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
- A61K2239/46—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the cancer treated
- A61K2239/48—Blood cells, e.g. leukemia or lymphoma
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862669848P | 2018-05-10 | 2018-05-10 | |
| US62/669,848 | 2018-05-10 | ||
| PCT/US2019/031699 WO2019217803A1 (en) | 2018-05-10 | 2019-05-10 | Gene therapy methods and compositions using auxotrophic regulatable cells |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN112513271A true CN112513271A (zh) | 2021-03-16 |
Family
ID=66794093
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980031608.1A Pending CN112513271A (zh) | 2018-05-10 | 2019-05-10 | 使用营养缺陷型可调控细胞进行基因治疗的方法和组合物 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20210052741A1 (https=) |
| EP (1) | EP3790974A1 (https=) |
| JP (1) | JP2021522865A (https=) |
| KR (1) | KR20210030902A (https=) |
| CN (1) | CN112513271A (https=) |
| AU (1) | AU2019265916A1 (https=) |
| BR (1) | BR112020022612A2 (https=) |
| CA (1) | CA3098874A1 (https=) |
| MX (1) | MX2020011757A (https=) |
| SG (1) | SG11202010837XA (https=) |
| WO (1) | WO2019217803A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20230265440A1 (en) * | 2020-06-26 | 2023-08-24 | The Board Of Trustees Of The Leland Stanford Junior University | Targeting the human ccr5 locus as a safe harbor for the expression of therapeutic proteins |
| WO2025080701A1 (en) * | 2023-10-11 | 2025-04-17 | William Marsh Rice University | Genetically activated mesenchymal stem cells and stromal cells |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016081924A1 (en) * | 2014-11-20 | 2016-05-26 | Duke University | Compositions, systems and methods for cell therapy |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030232430A1 (en) | 2001-11-26 | 2003-12-18 | Advanced Cell Technology | Methods for making and using reprogrammed human somatic cell nuclei and autologous and isogenic human stem cells |
| CA2608913A1 (en) * | 2005-05-18 | 2006-11-23 | Biofactura, Inc. | Compositions and methods for metabolic selection of transfected cells |
| US20090176260A1 (en) | 2007-12-04 | 2009-07-09 | Joseph Ching-Ming Wu | Double-fusion human embryonic stem cells, methods of making double-fusion human embryonic stem cells, triple-fusion human embryonic stem cells, methods of making triple-fusion human embryonic stem cells, and methods of monitoring double-fusion human embryonic stem cells and triple-fusion human embryonic stem cells |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| EP3363902B1 (en) | 2012-12-06 | 2019-11-27 | Sigma Aldrich Co. LLC | Crispr-based genome modification and regulation |
| CA2894684A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes |
| US20160122774A1 (en) | 2013-05-29 | 2016-05-05 | Cellectis | A method for producing precise dna cleavage using cas9 nickase activity |
| GB201618414D0 (en) * | 2016-11-01 | 2016-12-14 | Patterson James | Regulated cell lines and methods of use thereof |
-
2019
- 2019-05-10 WO PCT/US2019/031699 patent/WO2019217803A1/en not_active Ceased
- 2019-05-10 BR BR112020022612-2A patent/BR112020022612A2/pt not_active IP Right Cessation
- 2019-05-10 KR KR1020207035375A patent/KR20210030902A/ko not_active Withdrawn
- 2019-05-10 EP EP19729408.5A patent/EP3790974A1/en not_active Withdrawn
- 2019-05-10 CA CA3098874A patent/CA3098874A1/en active Pending
- 2019-05-10 MX MX2020011757A patent/MX2020011757A/es unknown
- 2019-05-10 SG SG11202010837XA patent/SG11202010837XA/en unknown
- 2019-05-10 JP JP2021513376A patent/JP2021522865A/ja not_active Withdrawn
- 2019-05-10 CN CN201980031608.1A patent/CN112513271A/zh active Pending
- 2019-05-10 AU AU2019265916A patent/AU2019265916A1/en not_active Abandoned
-
2020
- 2020-11-04 US US17/088,932 patent/US20210052741A1/en not_active Abandoned
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016081924A1 (en) * | 2014-11-20 | 2016-05-26 | Duke University | Compositions, systems and methods for cell therapy |
Non-Patent Citations (4)
| Title |
|---|
| HAN B ONG等: "Trypanosoma brucei (UMP synthase null mutants) are avirulent in mice, but recover virulence upon prolonged culture in vitro while retaining pyrimidine auxotrophy", 《MOL MICROBIOL.》, vol. 90, no. 2, pages 443 - 455, XP055607109, DOI: 10.1111/mmi.12376 * |
| KENTARO OH-HASHI等: "Application of a novel HiBiT peptide tag for monitoring ATF4 protein expression in Neuro2a cells", 《BIOCHEM BIOPHYS REP.》, vol. 12, pages 40 - 45, XP055607272, DOI: 10.1016/j.bbrep.2017.08.002 * |
| TAWNYA L BOWLES等: "Pancreatic cancer cell lines deficient in argininosuccinate synthetase are sensitive to arginine deprivation by arginine deiminase", 《INT J CANCER . 》, vol. 123, no. 8, pages 1950 - 5, XP055436309, DOI: 10.1002/ijc.23723 * |
| YOLANDA SANTIAGO 等: "Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases", 《PROC NATL ACAD SCI U S A .》, vol. 105, no. 15, pages 5809 - 14, XP009143037, DOI: 10.1073/pnas.0800940105 * |
Also Published As
| Publication number | Publication date |
|---|---|
| MX2020011757A (es) | 2021-01-08 |
| SG11202010837XA (en) | 2020-11-27 |
| BR112020022612A2 (pt) | 2021-02-09 |
| CA3098874A1 (en) | 2019-11-14 |
| US20210052741A1 (en) | 2021-02-25 |
| JP2021522865A (ja) | 2021-09-02 |
| WO2019217803A1 (en) | 2019-11-14 |
| AU2019265916A1 (en) | 2020-12-10 |
| EP3790974A1 (en) | 2021-03-17 |
| KR20210030902A (ko) | 2021-03-18 |
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