JP2020522269A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020522269A5 JP2020522269A5 JP2019567677A JP2019567677A JP2020522269A5 JP 2020522269 A5 JP2020522269 A5 JP 2020522269A5 JP 2019567677 A JP2019567677 A JP 2019567677A JP 2019567677 A JP2019567677 A JP 2019567677A JP 2020522269 A5 JP2020522269 A5 JP 2020522269A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- recombinant nucleic
- binding site
- mir
- mirna
- 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.)
- Pending
Links
- 108020004707 nucleic acids Proteins 0.000 claims 26
- 102000039446 nucleic acids Human genes 0.000 claims 26
- 150000007523 nucleic acids Chemical class 0.000 claims 26
- 230000027455 binding Effects 0.000 claims 23
- 108091070501 miRNA Proteins 0.000 claims 15
- 239000002679 microRNA Substances 0.000 claims 15
- 102000006890 Methyl-CpG-Binding Protein 2 Human genes 0.000 claims 9
- 108010072388 Methyl-CpG-Binding Protein 2 Proteins 0.000 claims 9
- 108091026890 Coding region Proteins 0.000 claims 8
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 5
- 108091028080 MiR-132 Proteins 0.000 claims 5
- 239000013598 vector Substances 0.000 claims 5
- 108091007780 MiR-122 Proteins 0.000 claims 4
- 108091028606 miR-1 stem-loop Proteins 0.000 claims 4
- 108091051828 miR-122 stem-loop Proteins 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 3
- 108090000565 Capsid Proteins Proteins 0.000 claims 3
- 102100023321 Ceruloplasmin Human genes 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 239000013603 viral vector Substances 0.000 claims 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 108020004705 Codon Proteins 0.000 claims 2
- 102000001708 Protein Isoforms Human genes 0.000 claims 2
- 108010029485 Protein Isoforms Proteins 0.000 claims 2
- 210000000234 capsid Anatomy 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 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
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 101001033726 Homo sapiens Methyl-CpG-binding protein 2 Proteins 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 230000008499 blood brain barrier function Effects 0.000 claims 1
- 210000001218 blood-brain barrier Anatomy 0.000 claims 1
- 102000049101 human MECP2 Human genes 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
- 230000009870 specific binding Effects 0.000 claims 1
- 230000035897 transcription Effects 0.000 claims 1
- 238000013518 transcription Methods 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241000701447 unidentified baculovirus Species 0.000 claims 1
- 241001529453 unidentified herpesvirus Species 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023135189A JP2023164447A (ja) | 2017-06-06 | 2023-08-23 | レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762516060P | 2017-06-06 | 2017-06-06 | |
| US62/516,060 | 2017-06-06 | ||
| PCT/US2018/036200 WO2018226785A1 (en) | 2017-06-06 | 2018-06-06 | Self-regulating aav vectors for safe expression of mecp2 in rett syndrome |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023135189A Division JP2023164447A (ja) | 2017-06-06 | 2023-08-23 | レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020522269A JP2020522269A (ja) | 2020-07-30 |
| JP2020522269A5 true JP2020522269A5 (cg-RX-API-DMAC7.html) | 2021-07-26 |
Family
ID=64567243
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019567677A Pending JP2020522269A (ja) | 2017-06-06 | 2018-06-06 | レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター |
| JP2023135189A Pending JP2023164447A (ja) | 2017-06-06 | 2023-08-23 | レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023135189A Pending JP2023164447A (ja) | 2017-06-06 | 2023-08-23 | レット症候群におけるｍｅｃｐ２の安全な発現のための自己調節ａａｖベクター |
Country Status (15)
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| WO2016131009A1 (en) | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| CN110325219B (zh) * | 2016-11-17 | 2024-03-26 | 全国儿童医院公司 | 编码甲基-CpG结合蛋白2的重组腺相关病毒的鞘内递送 |
| EP3574091B1 (en) | 2017-01-30 | 2023-02-22 | Brainvectis | Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias |
| EA202092362A1 (ru) | 2018-04-03 | 2021-01-11 | Страйдбайо, Инк. | Вирусные векторы для нацеливания на ткани глаза |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| AU2019354995B2 (en) | 2018-10-02 | 2024-12-12 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| US20220202960A1 (en) * | 2019-04-24 | 2022-06-30 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| CN114555813A (zh) * | 2019-05-21 | 2022-05-27 | 国家医疗保健研究所 | 治疗雷特综合征的胆固醇24-水解酶的表达载体 |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| WO2021113634A1 (en) * | 2019-12-05 | 2021-06-10 | The Board Of Regents Of The University Of Texas | Transgene cassettes designed to express a human mecp2 gene |
| US12497612B2 (en) | 2020-03-09 | 2025-12-16 | University Of Massachusetts | Gene replacement therapy for FOXG1 syndrome |
| PE20230767A1 (es) * | 2020-05-13 | 2023-05-09 | Voyager Therapeutics Inc | Redireccion del tropismo de las capsides de aav |
| AU2021300615A1 (en) * | 2020-06-30 | 2023-02-02 | The University Court Of The University Of Edinburgh | Transgene expression system |
| US12104163B2 (en) * | 2020-08-19 | 2024-10-01 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| CN113717248B (zh) * | 2020-09-30 | 2022-07-08 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2022094078A1 (en) * | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| IL311861A (en) | 2021-11-02 | 2024-06-01 | Voyager Therapeutics Inc | Aav capsid variants and uses thereof |
| GB202201242D0 (en) * | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| CN121219020A (zh) * | 2023-03-22 | 2025-12-26 | 圣拉斐尔医院有限责任公司 | 基因疗法 |
| CN121175427A (zh) * | 2023-05-15 | 2025-12-19 | 上海金珂博生物技术有限公司 | 一种Rett综合症的基因治疗 |
| TW202513796A (zh) * | 2023-06-09 | 2025-04-01 | 大陸商仁景（蘇州）生物科技有限公司 | 表達可調控的工程化ｒｎａ分子及其用途 |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| US20040106566A1 (en) | 2002-05-17 | 2004-06-03 | Shi-Lung Lin | RNA-splicing and processing-directed gene silencing and the relative applications thereof |
| BRPI0817812A2 (pt) | 2007-10-23 | 2015-04-14 | Novartis Ag | Uso de anticorpos trkb para o tratamento de distúrbios respiratórios |
| CN102015769B (zh) | 2008-01-17 | 2014-12-10 | Irm责任有限公司 | 改进的抗-trkb抗体 |
| WO2010105096A2 (en) | 2009-03-11 | 2010-09-16 | University Of Massachusetts | Modulation of human cytomegalovirus replication by micro-rna 132 (mir132), micro-rna 145 (mir145) and micro-rna 212 (mir212) |
| MX2013004577A (es) | 2010-10-25 | 2014-06-23 | Univ Aix Marseille Ii | Tratamiento de trastornos asociados a mecp2. |
| US20160000794A1 (en) | 2012-09-25 | 2016-01-07 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Serv | Methods for the diagnosis and treatment of sjogren's syndrome |
| WO2015038958A1 (en) | 2013-09-13 | 2015-03-19 | California Institute Of Technology | Selective recovery |
| WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| US10711270B2 (en) | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
| EP3209311B1 (en) | 2014-10-21 | 2024-03-06 | University of Massachusetts | Recombinant aav variants and uses thereof |
| EP3285780A4 (en) | 2015-04-24 | 2018-12-19 | University of Massachusetts | Modified aav constructions and uses thereof |
| EP3121284A1 (en) * | 2015-07-22 | 2017-01-25 | Ecole Polytechnique Fédérale de Lausanne (EPFL) | Bicistronic aav vector for rna interference in als |
-
2018
- 2018-06-06 EP EP18812887.0A patent/EP3635122A4/en active Pending
- 2018-06-06 MX MX2019014760A patent/MX2019014760A/es unknown
- 2018-06-06 US US16/619,733 patent/US11680275B2/en active Active
- 2018-06-06 BR BR112019025732-2A patent/BR112019025732A2/pt not_active IP Right Cessation
- 2018-06-06 EA EA201992882A patent/EA201992882A1/ru unknown
- 2018-06-06 AU AU2018281145A patent/AU2018281145A1/en not_active Abandoned
- 2018-06-06 KR KR1020207000120A patent/KR20200015701A/ko not_active Ceased
- 2018-06-06 JP JP2019567677A patent/JP2020522269A/ja active Pending
- 2018-06-06 CN CN201880051148.4A patent/CN111065742A/zh active Pending
- 2018-06-06 WO PCT/US2018/036200 patent/WO2018226785A1/en not_active Ceased
- 2018-06-06 SG SG11201911737PA patent/SG11201911737PA/en unknown
- 2018-06-06 CA CA3066623A patent/CA3066623A1/en active Pending
-
2019
- 2019-12-04 IL IL271170A patent/IL271170A/en unknown
- 2019-12-06 CL CL2019003582A patent/CL2019003582A1/es unknown
- 2019-12-27 CO CONC2019/0014840A patent/CO2019014840A2/es unknown
-
2023
- 2023-05-03 US US18/311,367 patent/US20230416779A1/en active Pending
- 2023-08-23 JP JP2023135189A patent/JP2023164447A/ja active Pending