JP2017510293A5 - - Google Patents
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- JP2017510293A5 JP2017510293A5 JP2016575630A JP2016575630A JP2017510293A5 JP 2017510293 A5 JP2017510293 A5 JP 2017510293A5 JP 2016575630 A JP2016575630 A JP 2016575630A JP 2016575630 A JP2016575630 A JP 2016575630A JP 2017510293 A5 JP2017510293 A5 JP 2017510293A5
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- JP
- Japan
- Prior art keywords
- seq
- vector
- composition
- pharmaceutical composition
- ctcf
- Prior art date
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- 239000012212 insulator Substances 0.000 claims description 78
- 102000016897 CCCTC-Binding Factor Human genes 0.000 claims description 60
- 108010014064 CCCTC-Binding Factor Proteins 0.000 claims description 60
- 239000013598 vector Substances 0.000 claims description 48
- 239000000203 mixture Substances 0.000 claims description 42
- 238000000034 method Methods 0.000 claims description 41
- 239000003623 enhancer Substances 0.000 claims description 15
- 239000003814 drug Substances 0.000 claims description 14
- 229940124597 therapeutic agent Drugs 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 13
- 230000000903 blocking effect Effects 0.000 claims description 11
- 239000013603 viral vector Substances 0.000 claims description 10
- 101100439689 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) chs-4 gene Proteins 0.000 claims description 9
- 230000001177 retroviral effect Effects 0.000 claims description 8
- 239000003795 chemical substances by application Substances 0.000 claims description 6
- 108091070501 miRNA Proteins 0.000 claims description 6
- 239000002679 microRNA Substances 0.000 claims description 6
- 102000004169 proteins and genes Human genes 0.000 claims description 6
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 4
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 4
- 206010020751 Hypersensitivity Diseases 0.000 claims description 4
- 108091023045 Untranslated Region Proteins 0.000 claims description 4
- 238000010367 cloning Methods 0.000 claims description 4
- 201000010099 disease Diseases 0.000 claims description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 4
- 230000000694 effects Effects 0.000 claims description 4
- 230000003584 silencer Effects 0.000 claims description 4
- 230000003612 virological effect Effects 0.000 claims description 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 3
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 claims description 3
- 230000009368 gene silencing by RNA Effects 0.000 claims description 3
- 230000002452 interceptive effect Effects 0.000 claims description 3
- 238000001353 Chip-sequencing Methods 0.000 claims description 2
- 206010028980 Neoplasm Diseases 0.000 claims description 2
- 238000001415 gene therapy Methods 0.000 claims description 2
- 102000039446 nucleic acids Human genes 0.000 claims description 2
- 108020004707 nucleic acids Proteins 0.000 claims description 2
- 150000007523 nucleic acids Chemical class 0.000 claims description 2
- 230000005740 tumor formation Effects 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 24
- 239000000126 substance Substances 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- 230000002068 genetic effect Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461953419P | 2014-03-14 | 2014-03-14 | |
| US61/953,419 | 2014-03-14 | ||
| US201462068226P | 2014-10-24 | 2014-10-24 | |
| US62/068,226 | 2014-10-24 | ||
| PCT/US2015/020369 WO2015138852A1 (en) | 2014-03-14 | 2015-03-13 | Genomic insulator elements and uses thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017510293A JP2017510293A (ja) | 2017-04-13 |
| JP2017510293A5 true JP2017510293A5 (enExample) | 2018-04-26 |
| JP6737714B2 JP6737714B2 (ja) | 2020-08-12 |
Family
ID=54072444
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016575630A Active JP6737714B2 (ja) | 2014-03-14 | 2015-03-13 | ゲノムインスレーターエレメントおよびその使用 |
Country Status (5)
| Country | Link |
|---|---|
| US (3) | US10590433B2 (enExample) |
| EP (1) | EP3117004A4 (enExample) |
| JP (1) | JP6737714B2 (enExample) |
| AU (1) | AU2015229227B2 (enExample) |
| WO (1) | WO2015138852A1 (enExample) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105408483A (zh) | 2013-03-15 | 2016-03-16 | 通用医疗公司 | 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向 |
| EP3117004A4 (en) * | 2014-03-14 | 2017-12-06 | University of Washington | Genomic insulator elements and uses thereof |
| BR112017004349A2 (pt) * | 2014-09-04 | 2017-12-05 | Memorial Sloan Kettering Cancer Center | terapia com gene de globina para o tratamento de hemoglobinopatias |
| KR102067352B1 (ko) * | 2015-11-24 | 2020-01-16 | 글락소스미스클라인 인털렉츄얼 프로퍼티 디벨로프먼트 리미티드 | 레트로바이러스 생산을 위한 일시적인 형질감염 방법 |
| EP3612204A4 (en) | 2017-04-21 | 2021-01-27 | The General Hospital Corporation | INDUCTIBLE, TUNING AND MULTIPLEX HUMAN GENE REGULATION USING CRISPR-CPF1 |
| US11970707B2 (en) | 2017-09-18 | 2024-04-30 | Children's Hospital Medical Center | Strong insulator and uses thereof in gene delivery |
| EP3720509A4 (en) | 2017-12-06 | 2021-12-08 | Memorial Sloan-Kettering Cancer Center | GENE THERAPY WITH A GLOBIN GENE FOR THE TREATMENT OF HEMOGLOBINOPATHIES |
| CA3100726A1 (en) | 2018-05-17 | 2019-11-21 | The General Hospital Corporation | Ccctc-binding factor variants |
| WO2020040046A1 (ja) * | 2018-08-21 | 2020-02-27 | 学校法人慶應義塾 | エンハンサーブロッカー及びその使用 |
| CN110747233A (zh) * | 2019-08-21 | 2020-02-04 | 常州市第二人民医院 | 含一种绝缘子的慢病毒稳定表达载体及其构建方法 |
| CN110760543B (zh) * | 2019-08-21 | 2021-12-10 | 常州市第二人民医院 | 一种运用绝缘子组合构建的慢病毒多启动子稳定表达载体及构建方法 |
| US12702720B2 (en) | 2020-05-14 | 2026-08-11 | St. Jude Children's Research Hospital, Inc. | Genomic insulator element exhibiting enhancer blocking activities in lymphocytes and uses thereof |
| WO2024201066A1 (en) | 2023-03-31 | 2024-10-03 | Dawn Therapeutics Limited | Novel vector |
| EP4790141A1 (en) * | 2023-10-08 | 2026-08-12 | Shanghai Qilu Pharmaceutical Research and Development Centre Ltd. | Truncated insulator and use thereof in transient expression of recombinant protein |
| EP4545644A1 (en) * | 2023-10-24 | 2025-04-30 | Ospedale San Raffaele S.r.l. | Polynucleotides |
| WO2025235425A1 (en) * | 2024-05-06 | 2025-11-13 | Research Institute At Nationwide Children's Hospital | Improved proviral plasmids |
| WO2026064752A1 (en) * | 2024-09-22 | 2026-03-26 | Newberry Skyler | Compositions and methods for in vivo genetic engineering of lymphocyte precursors |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5075109A (en) | 1986-10-24 | 1991-12-24 | Southern Research Institute | Method of potentiating an immune response |
| US4897268A (en) | 1987-08-03 | 1990-01-30 | Southern Research Institute | Drug delivery system and method of making the same |
| US5610053A (en) * | 1993-04-07 | 1997-03-11 | The United States Of America As Represented By The Department Of Health And Human Services | DNA sequence which acts as a chromatin insulator element to protect expressed genes from cis-acting regulatory sequences in mammalian cells |
| US5972643A (en) | 1994-06-17 | 1999-10-26 | Fred Hutchinson Cancer Research Center | Isolated polynucleotide molecules encoding CTCF, a CCCTC-binding factor |
| AU776752B2 (en) | 1999-06-30 | 2004-09-23 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | DNA binding protein and sequence as insulators having specific enhancer blocking activity for regulation of gene expression |
| WO2002088346A2 (en) | 2001-05-01 | 2002-11-07 | National Research Council Of Canada | A system for inducible expression in eukaryotic cells |
| US20050283854A1 (en) | 2003-05-23 | 2005-12-22 | Anton Krumm | Recombinant vectors for use in position-independent transgene expression within chromatin |
| WO2008101216A2 (en) | 2007-02-15 | 2008-08-21 | The Govt. Of The Usa As Represented By The Secretary Of The Dept Of Health And Human Services | Gamma satellite insulator sequences and their use in preventing gene silencing |
| WO2009134420A2 (en) | 2008-05-01 | 2009-11-05 | The Salk Institute For Biological Studies | Epigenetic silencing of tumor suppressor genes |
| US20110294873A1 (en) * | 2008-10-23 | 2011-12-01 | Université de Lausanne | Gene Transfer Vectors Comprising At Least One Isolated DNA Molecule Having Insulator Or Boundary Properties And Methods To Identify The Same |
| WO2010108143A2 (en) * | 2009-03-20 | 2010-09-23 | University Of Washington | Global mapping of protein-dna interaction by digital genomic footprinting |
| EP2414524B1 (en) * | 2009-04-03 | 2017-08-23 | Centre National De La Recherche Scientifique | Gene transfer vectors comprising genetic insulator elements and methods to identify genetic insulator elements |
| 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 |
| WO2013152352A1 (en) * | 2012-04-06 | 2013-10-10 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Servic | Live, attenuated rubella vector to express vaccine antigens |
| EP3117004A4 (en) * | 2014-03-14 | 2017-12-06 | University of Washington | Genomic insulator elements and uses thereof |
| BR112017004349A2 (pt) | 2014-09-04 | 2017-12-05 | Memorial Sloan Kettering Cancer Center | terapia com gene de globina para o tratamento de hemoglobinopatias |
-
2015
- 2015-03-13 EP EP15760641.9A patent/EP3117004A4/en active Pending
- 2015-03-13 JP JP2016575630A patent/JP6737714B2/ja active Active
- 2015-03-13 AU AU2015229227A patent/AU2015229227B2/en active Active
- 2015-03-13 WO PCT/US2015/020369 patent/WO2015138852A1/en not_active Ceased
- 2015-03-13 US US15/126,115 patent/US10590433B2/en active Active
-
2020
- 2020-01-31 US US16/778,996 patent/US11788101B2/en active Active
-
2023
- 2023-09-07 US US18/462,870 patent/US20240182924A1/en active Pending
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