JP2020522269A5 - - Google Patents
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- 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 (enExample) | 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)
| Country | Link |
|---|---|
| US (2) | US11680275B2 (enExample) |
| EP (1) | EP3635122A4 (enExample) |
| JP (2) | JP2020522269A (enExample) |
| KR (1) | KR20200015701A (enExample) |
| CN (1) | CN111065742A (enExample) |
| AU (1) | AU2018281145A1 (enExample) |
| BR (1) | BR112019025732A2 (enExample) |
| CA (1) | CA3066623A1 (enExample) |
| CL (1) | CL2019003582A1 (enExample) |
| CO (1) | CO2019014840A2 (enExample) |
| EA (1) | EA201992882A1 (enExample) |
| IL (1) | IL271170A (enExample) |
| MX (1) | MX2019014760A (enExample) |
| SG (1) | SG11201911737PA (enExample) |
| WO (1) | WO2018226785A1 (enExample) |
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 |
| CA3044291A1 (en) * | 2016-11-17 | 2018-05-24 | Nationwide Children's Hospital Inc. | Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2 |
| WO2018138371A1 (en) | 2017-01-30 | 2018-08-02 | Brainvectis | Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias |
| CN112272672A (zh) | 2018-04-03 | 2021-01-26 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| JP2021519581A (ja) | 2018-04-03 | 2021-08-12 | ストライドバイオ，インコーポレイテッド | 抗体を回避するウイルスベクター |
| KR20210006358A (ko) | 2018-04-03 | 2021-01-18 | 스트라이드바이오 인코포레이티드 | 안 조직을 표적으로 하기 위한 바이러스 벡터 |
| CA3115217A1 (en) | 2018-10-02 | 2020-04-09 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| EP3973060A4 (en) * | 2019-04-24 | 2023-05-10 | The Trustees of The University of Pennsylvania | COMPOSITIONS USEFUL FOR THE TREATMENT OF RETT SYNDROME |
| US20220233654A1 (en) * | 2019-05-21 | 2022-07-28 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome |
| US11905523B2 (en) | 2019-10-17 | 2024-02-20 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C |
| 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 |
| EP4117735A4 (en) * | 2020-03-09 | 2024-04-10 | University of Massachusetts | Gene replacement therapy for foxg1 syndrome |
| MX2022014256A (es) * | 2020-05-13 | 2023-01-11 | Voyager Therapeutics Inc | Redirección del tropismo de las cápsides de aav. |
| US20230323391A1 (en) * | 2020-06-30 | 2023-10-12 | The University Court Of The University Of Edinburgh | Transgene expression system |
| IL300728A (en) * | 2020-08-19 | 2023-04-01 | Sarepta Therapeutics Inc | Adeno-associated virus vectors for the 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 |
| TW202334436A (zh) | 2021-11-02 | 2023-09-01 | 美商航海家醫療公司 | Ａａｖ衣殼變異體及其用途 |
| GB202201242D0 (en) * | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| WO2024194491A1 (en) * | 2023-03-22 | 2024-09-26 | Ospedale San Raffaele S.R.L. | Gene therapy |
| 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 |
| CN119101681A (zh) * | 2023-06-09 | 2024-12-10 | 仁景(苏州)生物科技有限公司 | 表达可调控的工程化rna分子 |
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 |
| BR122016004546B8 (pt) | 2001-11-13 | 2021-07-27 | Univ Pennsylvania | vírus adenoassociado recombinante, método de geração do referido vírus e composição compreendendo o referido vírus |
| 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 |
| TW200936608A (en) | 2008-01-17 | 2009-09-01 | Irm Llc | Improved anti-TrkB antibodies |
| 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) |
| EA201390616A1 (ru) | 2010-10-25 | 2013-09-30 | Юниверсите Д'Экс-Марсель | ЛЕЧЕНИЕ НАРУШЕНИЙ, АССОЦИИРОВАННЫХ С ГЕНОМ 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 |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
| US10975391B2 (en) | 2014-04-25 | 2021-04-13 | University Of Massachusetts | Recombinant AAV vectors useful for reducing immunity against transgene products |
| JP6842410B2 (ja) | 2014-10-03 | 2021-03-17 | ユニバーシティ オブ マサチューセッツ | 新規の高効率ライブラリーにより同定されるａａｖベクター |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| JP7023108B2 (ja) | 2014-10-21 | 2022-02-21 | ユニバーシティ オブ マサチューセッツ | 組み換えａａｖバリアントおよびその使用 |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs 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 EA EA201992882A patent/EA201992882A1/ru unknown
- 2018-06-06 AU AU2018281145A patent/AU2018281145A1/en not_active Abandoned
- 2018-06-06 US US16/619,733 patent/US11680275B2/en active Active
- 2018-06-06 CN CN201880051148.4A patent/CN111065742A/zh active Pending
- 2018-06-06 KR KR1020207000120A patent/KR20200015701A/ko not_active Ceased
- 2018-06-06 JP JP2019567677A patent/JP2020522269A/ja active Pending
- 2018-06-06 BR BR112019025732-2A patent/BR112019025732A2/pt not_active IP Right Cessation
- 2018-06-06 EP EP18812887.0A patent/EP3635122A4/en active Pending
- 2018-06-06 MX MX2019014760A patent/MX2019014760A/es unknown
- 2018-06-06 SG SG11201911737PA patent/SG11201911737PA/en unknown
- 2018-06-06 CA CA3066623A patent/CA3066623A1/en active Pending
- 2018-06-06 WO PCT/US2018/036200 patent/WO2018226785A1/en not_active Ceased
-
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