JPWO2020084162A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020084162A5 JPWO2020084162A5 JP2021522959A JP2021522959A JPWO2020084162A5 JP WO2020084162 A5 JPWO2020084162 A5 JP WO2020084162A5 JP 2021522959 A JP2021522959 A JP 2021522959A JP 2021522959 A JP2021522959 A JP 2021522959A JP WO2020084162 A5 JPWO2020084162 A5 JP WO2020084162A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- expression cassette
- tfbs
- regulatory element
- 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.)
- Granted
Links
- 150000007523 nucleic acids Chemical class 0.000 claims 44
- 102000039446 nucleic acids Human genes 0.000 claims 42
- 108020004707 nucleic acids Proteins 0.000 claims 42
- 230000014509 gene expression Effects 0.000 claims 25
- 108091023040 Transcription factor Proteins 0.000 claims 22
- 102000040945 Transcription factor Human genes 0.000 claims 22
- 230000001105 regulatory effect Effects 0.000 claims 21
- 108700019146 Transgenes Proteins 0.000 claims 9
- 210000004185 liver Anatomy 0.000 claims 6
- 108010076282 Factor IX Proteins 0.000 claims 4
- 108010054218 Factor VIII Proteins 0.000 claims 4
- 102000001690 Factor VIII Human genes 0.000 claims 4
- 229960000301 factor viii Drugs 0.000 claims 4
- 239000012634 fragment Substances 0.000 claims 4
- 108010039209 Blood Coagulation Factors Proteins 0.000 claims 3
- 102000015081 Blood Coagulation Factors Human genes 0.000 claims 3
- 108010071690 Prealbumin Proteins 0.000 claims 3
- 239000003114 blood coagulation factor Substances 0.000 claims 3
- 210000005229 liver cell Anatomy 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 102100034798 CCAAT/enhancer-binding protein beta Human genes 0.000 claims 2
- 102100022641 Coagulation factor IX Human genes 0.000 claims 2
- 102100021429 DNA-directed RNA polymerase II subunit RPB1 Human genes 0.000 claims 2
- 101000945963 Homo sapiens CCAAT/enhancer-binding protein beta Proteins 0.000 claims 2
- 101001106401 Homo sapiens DNA-directed RNA polymerase II subunit RPB1 Proteins 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 102000009190 Transthyretin Human genes 0.000 claims 2
- 230000002708 enhancing effect Effects 0.000 claims 2
- 229960004222 factor ix Drugs 0.000 claims 2
- 238000001415 gene therapy Methods 0.000 claims 2
- 208000013403 hyperactivity Diseases 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 102100038511 AT-rich interactive domain-containing protein 3A Human genes 0.000 claims 1
- 102100034799 CCAAT/enhancer-binding protein delta Human genes 0.000 claims 1
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 102100028121 Fos-related antigen 2 Human genes 0.000 claims 1
- 108010081348 HRT1 protein Hairy Proteins 0.000 claims 1
- 102100021881 Hairy/enhancer-of-split related with YRPW motif protein 1 Human genes 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 102100029283 Hepatocyte nuclear factor 3-alpha Human genes 0.000 claims 1
- 102100029284 Hepatocyte nuclear factor 3-beta Human genes 0.000 claims 1
- 102100039999 Histone deacetylase 2 Human genes 0.000 claims 1
- 101000808887 Homo sapiens AT-rich interactive domain-containing protein 3A Proteins 0.000 claims 1
- 101000945965 Homo sapiens CCAAT/enhancer-binding protein delta Proteins 0.000 claims 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 1
- 101001059934 Homo sapiens Fos-related antigen 2 Proteins 0.000 claims 1
- 101001062353 Homo sapiens Hepatocyte nuclear factor 3-alpha Proteins 0.000 claims 1
- 101001062347 Homo sapiens Hepatocyte nuclear factor 3-beta Proteins 0.000 claims 1
- 101001035011 Homo sapiens Histone deacetylase 2 Proteins 0.000 claims 1
- 101000593405 Homo sapiens Myb-related protein B Proteins 0.000 claims 1
- 101001093899 Homo sapiens Retinoic acid receptor RXR-alpha Proteins 0.000 claims 1
- 101001050297 Homo sapiens Transcription factor JunD Proteins 0.000 claims 1
- 101000759547 Homo sapiens Zinc finger and BTB domain-containing protein 7A Proteins 0.000 claims 1
- 241000702623 Minute virus of mice Species 0.000 claims 1
- 102100034670 Myb-related protein B Human genes 0.000 claims 1
- 102100035178 Retinoic acid receptor RXR-alpha Human genes 0.000 claims 1
- 102100023118 Transcription factor JunD Human genes 0.000 claims 1
- 102100023264 Zinc finger and BTB domain-containing protein 7A Human genes 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 229940105778 coagulation factor viii Drugs 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 230000002163 immunogen Effects 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
- 230000005030 transcription termination Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18202986.8 | 2018-10-26 | ||
| EP18202986 | 2018-10-26 | ||
| PCT/EP2019/079358 WO2020084162A1 (en) | 2018-10-26 | 2019-10-28 | Liver-specific nucleic acid regulatory elements and methods and use thereof |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022505877A JP2022505877A (ja) | 2022-01-14 |
| JPWO2020084162A5 true JPWO2020084162A5 (https=) | 2022-11-08 |
| JP2022505877A5 JP2022505877A5 (https=) | 2022-11-08 |
| JP7529286B2 JP7529286B2 (ja) | 2024-08-06 |
Family
ID=64316267
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021522959A Active JP7529286B2 (ja) | 2018-10-26 | 2019-10-28 | 肝特異的核酸調節エレメント並びにその方法及び使用 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20210403948A1 (https=) |
| EP (1) | EP3870237A1 (https=) |
| JP (1) | JP7529286B2 (https=) |
| CN (1) | CN112912112B (https=) |
| WO (1) | WO2020084162A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3823985A1 (en) | 2018-07-16 | 2021-05-26 | Baxalta Incorporated | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| TWI851647B (zh) * | 2019-01-16 | 2024-08-11 | 日商武田藥品工業股份有限公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體 |
| CN110791502B (zh) * | 2019-12-04 | 2022-07-08 | 陕西理工大学 | 人源rbp4启动子片段及其应用 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| AU2022242921A1 (en) * | 2021-03-25 | 2023-10-19 | The Johns Hopkins University | Hydrodynamic gene delivery |
| JP2025534900A (ja) * | 2022-10-08 | 2025-10-21 | リンイ バイオテック カンパニー リミテッド | 遺伝子発現を増強するためのコンストラクト |
| WO2025092951A1 (en) * | 2023-11-03 | 2025-05-08 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant adeno-associated viral vectors for treating fabry disease |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7351813B2 (en) | 2000-06-20 | 2008-04-01 | The Board Of Trustees Of The Leland Stanford Junior University | Liver-specific gene expression cassettes, and methods of use |
| US20070161031A1 (en) * | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
| US20090018031A1 (en) * | 2006-12-07 | 2009-01-15 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways tools, and methods |
| CN102618536B (zh) * | 2008-02-14 | 2013-10-16 | 财团法人牧岩生命工学研究所 | 适于表达用于基因治疗的编码序列的表达载体 |
| EP4154903A1 (en) | 2008-04-22 | 2023-03-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| US20100323376A1 (en) * | 2009-06-17 | 2010-12-23 | Maine Standards Company, Llc | Method for Measuring Lipoprotein-Specific Apolipoproteins |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| US8815779B2 (en) * | 2009-09-16 | 2014-08-26 | SwitchGear Genomics, Inc. | Transcription biomarkers of biological responses and methods |
| WO2011051450A1 (en) | 2009-10-29 | 2011-05-05 | Vib Vzw | Cardiac-specific nucleic acid regulatory elements and methods and use thereof |
| US20150141320A1 (en) * | 2012-05-16 | 2015-05-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| WO2013186398A1 (en) * | 2012-06-15 | 2013-12-19 | Fondazione Telethon | Tfeb for use in treating obesity or metabolic syndrome |
| EP2911687B1 (en) | 2012-10-26 | 2019-02-13 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| KR101429696B1 (ko) * | 2012-11-21 | 2014-08-13 | 국립암센터 | 안전성 및 항암활성이 증가된 재조합 아데노바이러스 및 이의 용도 |
| EP3270944B1 (en) | 2015-03-17 | 2019-10-23 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
| CA3002524A1 (en) * | 2015-10-28 | 2017-05-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
-
2019
- 2019-10-28 US US17/288,425 patent/US20210403948A1/en active Pending
- 2019-10-28 EP EP19789570.9A patent/EP3870237A1/en active Pending
- 2019-10-28 WO PCT/EP2019/079358 patent/WO2020084162A1/en not_active Ceased
- 2019-10-28 CN CN201980070613.3A patent/CN112912112B/zh active Active
- 2019-10-28 JP JP2021522959A patent/JP7529286B2/ja active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP3126348B2 (ja) | 組換えにより生産されたヒトｌｈ | |
| US5563045A (en) | Chimeric procoagulant proteins | |
| AU2013336601B2 (en) | Vector for liver-directed gene therapy of hemophilia and methods and use thereof | |
| JPS63500843A (ja) | 組換え繊維芽細胞成長因子 | |
| JP5367905B2 (ja) | ＤＬＫ１−Ｆｃ融合タンパク質を有効成分として含む癌転移抑制用組成物 | |
| JPH09511143A (ja) | アルファーウイルスｃＤＮＡベクター | |
| JP2002503459A (ja) | 繊維置換タンパク質を含有する修飾されたアデノウィルス | |
| US20210403948A1 (en) | Liver-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof | |
| US5859223A (en) | Soluble CR1 derivatives | |
| JPH11503910A (ja) | アデノウイルスヘルパーウイルスシステム | |
| JPWO2020084162A5 (https=) | ||
| WO2025189685A1 (zh) | 一种嵌合抗原受体纳米复合物及其制备方法和在制备抗衰老药物中的应用 | |
| KR101756131B1 (ko) | 간세포 성장인자 및 기질세포 유발인자1알파를 이용한 말초동맥질환의 예방 또는 치료용 조성물 | |
| JP7745307B2 (ja) | 連結ペプチド、連結ペプチドを含む血液凝固ｖｉｉｉ因子タンパク質又はその変異体及びその用途 | |
| MXPA06012321A (es) | Derivados c3 del complemento humano con funcion similar al factor de veneno de cobra. | |
| JPH09512422A (ja) | カイメース阻害活性を有するα−１−抗キモトリプシン類似体 | |
| JPH11178A (ja) | ヒトエンドグリン遺伝子プロモーターおよびその使用 | |
| KR102114880B1 (ko) | 코돈 최적화된 TIF1γ 폴리뉴클레오티드를 포함하는 재조합 벡터 및 이의 용도 | |
| CN116004725A (zh) | 间充质干细胞在疼痛相关疾病中的应用 | |
| JP7229519B2 (ja) | 血管新生促進剤 | |
| WO2023063842A1 (ru) | Нуклеотидная последовательность, кодирующая слитый белок | |
| JPWO2020038473A5 (https=) | ||
| CA2205078A1 (en) | Novel adenoviral vector for transferring human genes in vivo | |
| CN115678912B (zh) | 一种构建dgtm转基因斑马鱼作为载体表达人源dgtm蛋白的方法 | |
| WO2026017126A1 (zh) | 一种人工设计的新型nrg-1在疾病治疗中的应用 |