JPWO2020069339A5 - - Google Patents
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- JPWO2020069339A5 JPWO2020069339A5 JP2021517702A JP2021517702A JPWO2020069339A5 JP WO2020069339 A5 JPWO2020069339 A5 JP WO2020069339A5 JP 2021517702 A JP2021517702 A JP 2021517702A JP 2021517702 A JP2021517702 A JP 2021517702A JP WO2020069339 A5 JPWO2020069339 A5 JP WO2020069339A5
- Authority
- JP
- Japan
- Prior art keywords
- optionally
- nucleic acid
- sequence
- seq
- engineered nucleic
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 150000007523 nucleic acids Chemical class 0.000 claims 47
- 108020004707 nucleic acids Proteins 0.000 claims 41
- 102000039446 nucleic acids Human genes 0.000 claims 41
- 239000004098 Tetracycline Substances 0.000 claims 13
- 241000700605 Viruses Species 0.000 claims 13
- 229960002180 tetracycline Drugs 0.000 claims 13
- 229930101283 tetracycline Natural products 0.000 claims 13
- 235000019364 tetracycline Nutrition 0.000 claims 13
- 150000003522 tetracyclines Chemical class 0.000 claims 13
- 239000013598 vector Substances 0.000 claims 11
- 239000013603 viral vector Substances 0.000 claims 11
- 210000004027 cell Anatomy 0.000 claims 10
- 239000008194 pharmaceutical composition Substances 0.000 claims 10
- 230000035772 mutation Effects 0.000 claims 9
- 108700019146 Transgenes Proteins 0.000 claims 8
- 108700007698 Genetic Terminator Regions Proteins 0.000 claims 6
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 5
- 108090000765 processed proteins & peptides Proteins 0.000 claims 5
- 210000001519 tissue Anatomy 0.000 claims 5
- 108020004684 Internal Ribosome Entry Sites Proteins 0.000 claims 4
- 241001529453 unidentified herpesvirus Species 0.000 claims 4
- 241000710929 Alphavirus Species 0.000 claims 3
- 241000713666 Lentivirus Species 0.000 claims 3
- 241000700618 Vaccinia virus Species 0.000 claims 3
- 230000001939 inductive effect Effects 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 241000701161 unidentified adenovirus Species 0.000 claims 3
- 241001430294 unidentified retrovirus Species 0.000 claims 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 2
- -1 CAG Proteins 0.000 claims 2
- 108020004414 DNA Proteins 0.000 claims 2
- 210000000481 breast Anatomy 0.000 claims 2
- 210000003527 eukaryotic cell Anatomy 0.000 claims 2
- 239000013604 expression vector Substances 0.000 claims 2
- 230000014509 gene expression Effects 0.000 claims 2
- 238000010363 gene targeting Methods 0.000 claims 2
- 210000000214 mouth Anatomy 0.000 claims 2
- 210000003205 muscle Anatomy 0.000 claims 2
- 210000001236 prokaryotic cell Anatomy 0.000 claims 2
- 230000001737 promoting effect Effects 0.000 claims 2
- 230000001177 retroviral effect Effects 0.000 claims 2
- 102200001731 rs139203363 Human genes 0.000 claims 2
- 102220290202 rs1555242247 Human genes 0.000 claims 2
- 102220028424 rs386352293 Human genes 0.000 claims 2
- 230000001225 therapeutic effect Effects 0.000 claims 2
- 230000017423 tissue regeneration Effects 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- SGKRLCUYIXIAHR-AKNGSSGZSA-N (4s,4ar,5s,5ar,6r,12ar)-4-(dimethylamino)-1,5,10,11,12a-pentahydroxy-6-methyl-3,12-dioxo-4a,5,5a,6-tetrahydro-4h-tetracene-2-carboxamide Chemical compound C1=CC=C2[C@H](C)[C@@H]([C@H](O)[C@@H]3[C@](C(O)=C(C(N)=O)C(=O)[C@H]3N(C)C)(O)C3=O)C3=C(O)C2=C1O SGKRLCUYIXIAHR-AKNGSSGZSA-N 0.000 claims 1
- 101500011733 Acipenser gueldenstaedtii Acipensin 5 Proteins 0.000 claims 1
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 101100118093 Drosophila melanogaster eEF1alpha2 gene Proteins 0.000 claims 1
- 101000756632 Homo sapiens Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 101000579123 Homo sapiens Phosphoglycerate kinase 1 Proteins 0.000 claims 1
- KJWZYMMLVHIVSU-IYCNHOCDSA-N PGK1 Chemical compound CCCCC[C@H](O)\C=C\[C@@H]1[C@@H](CCCCCCC(O)=O)C(=O)CC1=O KJWZYMMLVHIVSU-IYCNHOCDSA-N 0.000 claims 1
- 102100028251 Phosphoglycerate kinase 1 Human genes 0.000 claims 1
- 101710182846 Polyhedrin Proteins 0.000 claims 1
- 101100010928 Saccharolobus solfataricus (strain ATCC 35092 / DSM 1617 / JCM 11322 / P2) tuf gene Proteins 0.000 claims 1
- 101150001810 TEAD1 gene Proteins 0.000 claims 1
- 101150074253 TEF1 gene Proteins 0.000 claims 1
- 108091023040 Transcription factor Proteins 0.000 claims 1
- 102000040945 Transcription factor Human genes 0.000 claims 1
- 102100029898 Transcriptional enhancer factor TEF-1 Human genes 0.000 claims 1
- 101150109071 UBC gene Proteins 0.000 claims 1
- 210000004102 animal cell Anatomy 0.000 claims 1
- 230000001580 bacterial effect Effects 0.000 claims 1
- 210000004204 blood vessel Anatomy 0.000 claims 1
- 210000000988 bone and bone Anatomy 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 102220402997 c.12G>T Human genes 0.000 claims 1
- 230000008668 cellular reprogramming Effects 0.000 claims 1
- 210000003555 cloaca Anatomy 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 229960003722 doxycycline Drugs 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 210000003238 esophagus Anatomy 0.000 claims 1
- 230000002538 fungal effect Effects 0.000 claims 1
- 102000009543 guanyl-nucleotide exchange factor activity proteins Human genes 0.000 claims 1
- 108040001860 guanyl-nucleotide exchange factor activity proteins Proteins 0.000 claims 1
- 210000000936 intestine Anatomy 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000004072 lung Anatomy 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 210000004165 myocardium Anatomy 0.000 claims 1
- 210000001331 nose Anatomy 0.000 claims 1
- 210000000056 organ Anatomy 0.000 claims 1
- 230000033667 organ regeneration Effects 0.000 claims 1
- 210000001672 ovary Anatomy 0.000 claims 1
- 210000000496 pancreas Anatomy 0.000 claims 1
- 210000003899 penis Anatomy 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 210000002307 prostate Anatomy 0.000 claims 1
- 230000009758 senescence Effects 0.000 claims 1
- 210000003491 skin Anatomy 0.000 claims 1
- 210000002784 stomach Anatomy 0.000 claims 1
- 238000012360 testing method Methods 0.000 claims 1
- 210000001550 testis Anatomy 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862738894P | 2018-09-28 | 2018-09-28 | |
| US62/738,894 | 2018-09-28 | ||
| PCT/US2019/053492 WO2020069339A1 (en) | 2018-09-28 | 2019-09-27 | Mutant reverse tetracycline transactivators for expression of genes |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022501060A JP2022501060A (ja) | 2022-01-06 |
| JPWO2020069339A5 true JPWO2020069339A5 (https=) | 2022-10-05 |
| JP2022501060A5 JP2022501060A5 (https=) | 2022-10-05 |
| JP7589997B2 JP7589997B2 (ja) | 2024-11-26 |
Family
ID=68242854
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021517702A Active JP7589997B2 (ja) | 2018-09-28 | 2019-09-27 | 遺伝子の発現のための変異体リバーステトラサイクリントランスアクチベーター |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20210403923A1 (https=) |
| EP (1) | EP3856260A1 (https=) |
| JP (1) | JP7589997B2 (https=) |
| CN (1) | CN113164625B (https=) |
| CA (1) | CA3113470A1 (https=) |
| WO (1) | WO2020069339A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12274733B2 (en) | 2018-09-28 | 2025-04-15 | President And Fellows Of Harvard College | Cellular reprogramming to reverse aging and promote organ and tissue regeneration |
| MX2021003663A (es) | 2018-09-28 | 2021-05-28 | Harvard College | Reprogramacion celular para revertir el envejecimiento y promover la regeneracion de organos y tejidos. |
| WO2021091935A1 (en) * | 2019-11-05 | 2021-05-14 | Lonza Walkersville, Inc. | Allogeneic t-cells and methods for production thereof |
| GB202103327D0 (en) * | 2021-03-10 | 2021-04-21 | Babraham Inst | Novel method |
| US12467917B2 (en) | 2021-03-16 | 2025-11-11 | California Institute Of Technology | Reconstructing human early embryogenesis in vitro with pluripotent stem cells |
| EP4355882A2 (en) * | 2021-06-15 | 2024-04-24 | Modernatx, Inc. | Engineered polynucleotides for cell-type or microenvironment-specific expression |
| JP2025511883A (ja) * | 2022-04-06 | 2025-04-16 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 中枢神経系の加齢を逆行させること |
| CN119546324A (zh) * | 2022-07-12 | 2025-02-28 | 南京瑞初医药有限公司 | 组合物及其使用方法 |
| US20260035707A1 (en) * | 2022-07-26 | 2026-02-05 | Modernatx, Inc. | Engineered polynucleotides for temporal control of expression |
| CN116004789B (zh) * | 2022-08-01 | 2024-06-04 | 湖南家辉生物技术有限公司 | 一种Seckel综合征致病基因CEP152突变位点的应用及其诊断试剂 |
| US12252518B2 (en) | 2023-01-06 | 2025-03-18 | Life Biosciences, Inc. | Methods of treating non-arteritic anterior ischemic optic neuropathy |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5549910A (en) | 1989-03-31 | 1996-08-27 | The Regents Of The University Of California | Preparation of liposome and lipid complex compositions |
| JP3218637B2 (ja) | 1990-07-26 | 2001-10-15 | 大正製薬株式会社 | 安定なリポソーム水懸濁液 |
| JP2958076B2 (ja) | 1990-08-27 | 1999-10-06 | 株式会社ビタミン研究所 | 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法 |
| US5399363A (en) | 1991-01-25 | 1995-03-21 | Eastman Kodak Company | Surface modified anticancer nanoparticles |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| US5741516A (en) | 1994-06-20 | 1998-04-21 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| AU746850B2 (en) * | 1994-07-01 | 2002-05-02 | Tet Systems Holding Gmbh & Co. Kg | Tetracycline-regulated transcriptional modulaters |
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| US5738868A (en) | 1995-07-18 | 1998-04-14 | Lipogenics Ltd. | Liposome compositions and kits therefor |
| DE19851415B4 (de) * | 1998-11-07 | 2010-11-04 | Wolfgang Hillen | Mutanter Transaktivator |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| US20030186281A1 (en) * | 2001-12-21 | 2003-10-02 | Wolfgang Hillen | Modified tetracycline repressor protein compositions and methods of use |
| GB0327384D0 (en) * | 2003-11-25 | 2003-12-31 | Queen Mary & Westfield College | Gene therapy |
| JP5155176B2 (ja) * | 2005-11-17 | 2013-02-27 | ティーイーティー システムズ ゲーエムベーハー ウント ツェーオー． カーゲー | 誘導発現系 |
| WO2008051854A2 (en) * | 2006-10-20 | 2008-05-02 | Trustees Of Boston University | A tunable genetic switch for regulating gene expression |
| US8080647B2 (en) * | 2006-11-22 | 2011-12-20 | Pioneer Hi Bred International Inc | Tetracycline repressor and uses thereof |
| CN101302553A (zh) * | 2008-05-30 | 2008-11-12 | 广州华峰生物科技有限公司 | 基于环介导等温扩增技术的结核分歧杆菌基因快速诊断试剂盒及其检测方法 |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| EP2643459B1 (en) * | 2010-11-24 | 2017-09-27 | Takara Bio USA, Inc. | Inducible expression system transcription modulators comprising a distributed protein transduction domain and methods for using the same |
| EP2710130B1 (en) * | 2011-05-19 | 2022-06-29 | Fundación Pública Andaluza Progreso Y Salud | Highly inducible dual-promoter lentiviral tet-on system |
| CN102816793B (zh) * | 2012-08-22 | 2014-09-10 | 陕西师范大学 | 单载体双向启动的Tet-on诱导表达系统及其构建方法和应用 |
| DK2898076T3 (en) * | 2012-09-19 | 2018-06-14 | Dsm Ip Assets Bv | METHOD OF CELL MODIFICATION USING ESSENTIAL GENES AS MARKERS AND EVEN RECYCLING THESE |
| KR20150087321A (ko) * | 2012-11-21 | 2015-07-29 | 메르츠 파마 게엠베하 운트 코. 카가아 | 보툴리눔 신경독소 생물학적 활성의 결정을 위한 수단 및 방법 |
| WO2014152607A2 (en) * | 2013-03-14 | 2014-09-25 | University Of Massachusetts | Dosage compensating transgenes and cells |
| EP3298141A4 (en) * | 2015-05-17 | 2019-01-09 | The Medical Research, Infrastructure, And Health Services Fund Of The Tel Aviv Medical Center | COMPOSITIONS AND METHOD FOR THE TREATMENT OF CANCER |
| AU2016315703A1 (en) * | 2015-08-31 | 2018-03-08 | The Trustees Of The University Of Pennsylvania | Chimeric AAV-anti-VEGF for treating cancer in canines |
| WO2019023680A1 (en) * | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| WO2019099552A1 (en) * | 2017-11-14 | 2019-05-23 | The J. David Gladstone Institutes | Methods of generating retinal pigment epithelium (rpe) |
-
2019
- 2019-09-27 CN CN201980076921.7A patent/CN113164625B/zh active Active
- 2019-09-27 US US17/280,294 patent/US20210403923A1/en active Pending
- 2019-09-27 CA CA3113470A patent/CA3113470A1/en active Pending
- 2019-09-27 WO PCT/US2019/053492 patent/WO2020069339A1/en not_active Ceased
- 2019-09-27 JP JP2021517702A patent/JP7589997B2/ja active Active
- 2019-09-27 EP EP19787521.4A patent/EP3856260A1/en active Pending
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