JP2019524090A5 - - Google Patents
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- Publication number
- JP2019524090A5 JP2019524090A5 JP2019500531A JP2019500531A JP2019524090A5 JP 2019524090 A5 JP2019524090 A5 JP 2019524090A5 JP 2019500531 A JP2019500531 A JP 2019500531A JP 2019500531 A JP2019500531 A JP 2019500531A JP 2019524090 A5 JP2019524090 A5 JP 2019524090A5
- Authority
- JP
- Japan
- Prior art keywords
- rdh12
- composition
- functional
- nucleic acid
- acid sequence
- 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
- 101000742938 Homo sapiens Retinol dehydrogenase 12 Proteins 0.000 claims 22
- 102100038054 Retinol dehydrogenase 12 Human genes 0.000 claims 20
- 150000007523 nucleic acids Chemical group 0.000 claims 11
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 10
- 210000004027 cell Anatomy 0.000 claims 6
- 210000000234 capsid Anatomy 0.000 claims 5
- 108090000623 proteins and genes Proteins 0.000 claims 5
- 210000000608 photoreceptor cell Anatomy 0.000 claims 4
- 230000001105 regulatory effect Effects 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 239000003937 drug carrier Substances 0.000 claims 3
- 239000012634 fragment Substances 0.000 claims 3
- 108090000565 Capsid Proteins Proteins 0.000 claims 2
- 102100023321 Ceruloplasmin Human genes 0.000 claims 2
- 108020004705 Codon Proteins 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 201000010480 Leber congenital amaurosis 13 Diseases 0.000 claims 2
- 208000036575 RDH12-related recessive retinopathy Diseases 0.000 claims 2
- 208000030533 eye disease Diseases 0.000 claims 2
- 102000046986 human RDH12 Human genes 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 108091008695 photoreceptors Proteins 0.000 claims 2
- 239000013612 plasmid Substances 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 239000013598 vector Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 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
- 201000004569 Blindness Diseases 0.000 claims 1
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 1
- 101150033365 Rdh12 gene Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 238000003205 genotyping method Methods 0.000 claims 1
- 238000003384 imaging method Methods 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 230000001566 pro-viral effect Effects 0.000 claims 1
- 230000000717 retained effect Effects 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
- 230000004393 visual impairment Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022076693A JP7572990B2 (ja) | 2016-07-08 | 2022-05-06 | Ｒｄｈ１２が関与する疾患及び疾病を治療する方法及び組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662359777P | 2016-07-08 | 2016-07-08 | |
| US62/359,777 | 2016-07-08 | ||
| PCT/US2017/041122 WO2018009814A1 (en) | 2016-07-08 | 2017-07-07 | Methods and compositions for treatment of disorders and diseases involving rdh12 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022076693A Division JP7572990B2 (ja) | 2016-07-08 | 2022-05-06 | Ｒｄｈ１２が関与する疾患及び疾病を治療する方法及び組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019524090A JP2019524090A (ja) | 2019-09-05 |
| JP2019524090A5 true JP2019524090A5 (enExample) | 2020-07-16 |
| JP7101656B2 JP7101656B2 (ja) | 2022-07-15 |
Family
ID=60901399
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019500531A Active JP7101656B2 (ja) | 2016-07-08 | 2017-07-07 | Ｒｄｈ１２が関与する疾患及び疾病を治療する方法及び組成物 |
| JP2022076693A Active JP7572990B2 (ja) | 2016-07-08 | 2022-05-06 | Ｒｄｈ１２が関与する疾患及び疾病を治療する方法及び組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022076693A Active JP7572990B2 (ja) | 2016-07-08 | 2022-05-06 | Ｒｄｈ１２が関与する疾患及び疾病を治療する方法及び組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US11197936B2 (enExample) |
| EP (1) | EP3481958A4 (enExample) |
| JP (2) | JP7101656B2 (enExample) |
| KR (2) | KR102526506B1 (enExample) |
| CN (1) | CN110191954B (enExample) |
| AU (2) | AU2017292010B2 (enExample) |
| BR (1) | BR112019000171A2 (enExample) |
| CA (1) | CA3029646A1 (enExample) |
| IL (3) | IL297095B2 (enExample) |
| RU (1) | RU2764920C2 (enExample) |
| WO (1) | WO2018009814A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7261583B2 (ja) * | 2015-09-24 | 2023-04-20 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 補体媒介性疾患を処置するための組成物及び方法 |
| WO2018009814A1 (en) * | 2016-07-08 | 2018-01-11 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving rdh12 |
| WO2018204844A1 (en) | 2017-05-04 | 2018-11-08 | Lehigh University | Additive manufacturing system with tunable material properties |
| MY204271A (en) | 2017-11-15 | 2024-08-20 | Ucl Business Ltd | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Tern Therapeutics, Llc | Gene therapy for neuronal ceroid lipofuscinoses |
| TR2021022284A2 (tr) * | 2021-12-31 | 2022-01-21 | T C Ueskuedar Ueniversitesi | Retinitis pigmentosa (rp) hastalığının tedavisinde kullanılmak üzere retinol dehidrojenaz 12 (rdh12) gen mutasyonlarına yönelik crıspr-pe sistemi. |
| CN115725659A (zh) * | 2022-04-01 | 2023-03-03 | 首都医科大学附属北京同仁医院 | 一种应用视网膜原位基因编辑制作非人灵长类动物先天性黑矇模型的方法 |
| JP7168814B1 (ja) | 2022-07-01 | 2022-11-09 | 東洋インキＳｃホールディングス株式会社 | 積層体の分離回収方法 |
| CN118453915A (zh) * | 2024-04-29 | 2024-08-09 | 中国医学科学院北京协和医院 | 一种针对rdh12突变的基因治疗药物 |
Family Cites Families (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| ES2224375T3 (es) | 1997-04-14 | 2005-03-01 | Cell Genesys, Inc. | Metodos para aumentar la eficacia del producto de aav recombinante. |
| AU9397098A (en) | 1997-09-19 | 1999-04-12 | Trustees Of The University Of Pennsylvania, The | Methods and cell line useful for production of recombinant adeno-associated viruses |
| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
| CA2348382C (en) | 1998-11-10 | 2013-09-17 | The University Of North Carolina At Chapel Hill | Chimeric parvovirus vectors and methods of making and administering the same |
| EP2369002A1 (en) | 1999-08-09 | 2011-09-28 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| ES2327609T3 (es) | 2000-06-01 | 2009-11-02 | University Of North Carolina At Chapel Hill | Procedimientos y compuestos para controlar la liberacion de vectores de parvovirus reconbinantes. |
| WO2002082904A2 (en) | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| NZ618298A (en) | 2001-11-13 | 2015-04-24 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| JP4810062B2 (ja) | 2001-12-17 | 2011-11-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス（ａａｖ）血清型８の配列 |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| PT2311848E (pt) | 2002-12-23 | 2013-10-03 | Vical Inc | Vacinas à base de polinucleótido optimizadas por codão contra a infecção do citomegalovírus humano |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| HUE035567T2 (en) | 2003-09-30 | 2018-05-28 | Univ Pennsylvania | Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them |
| US7422840B2 (en) | 2004-11-12 | 2008-09-09 | E.I. Du Pont De Nemours And Company | Apparatus and process for forming a printing form having a cylindrical support |
| EP1866422B1 (en) | 2005-04-07 | 2016-04-06 | The Trustees of The University of Pennsylvania | Method of increasing the function of an aav vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20100081707A1 (en) | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| US7561973B1 (en) | 2008-07-31 | 2009-07-14 | Dna Twopointo, Inc. | Methods for determining properties that affect an expression property value of polynucleotides in an expression system |
| US7561972B1 (en) | 2008-06-06 | 2009-07-14 | Dna Twopointo, Inc. | Synthetic nucleic acids for expression of encoded proteins |
| US20120172419A1 (en) | 2009-09-15 | 2012-07-05 | Medical College Of Wisconsin Research Foundation Inc. | Reagents and methods for modulating cone photoreceptor activity |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| PT3486320T (pt) * | 2010-04-23 | 2022-03-30 | Univ Florida | Composições de ciclase raav-guanilato e métodos para tratar a amaurose congénita 1 de leber (acl1) |
| WO2012158757A1 (en) | 2011-05-16 | 2012-11-22 | The Trustees Of The University Of Pennsylvania | Proviral plasmids for production of recombinant adeno-associated virus |
| WO2013151663A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| AU2013287281B2 (en) | 2012-07-11 | 2018-04-26 | The Trustees Of The University Of Pennsylvania | AAV-mediated gene therapy for RPGR x-linked retinal degeneration |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| US8962449B1 (en) | 2013-07-30 | 2015-02-24 | Micron Technology, Inc. | Methods for processing semiconductor devices |
| CA2930282C (en) * | 2013-11-20 | 2024-09-10 | Fond Telethon | Artificial dna-binding proteins and uses thereof |
| KR102288849B1 (ko) | 2014-03-17 | 2021-08-12 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| PT3137497T (pt) | 2014-05-02 | 2021-07-12 | Genzyme Corp | Vetores de aav para terapia genética na retina e snc |
| WO2016019364A1 (en) * | 2014-08-01 | 2016-02-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for self-regulated inducible gene expression |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| WO2018009814A1 (en) | 2016-07-08 | 2018-01-11 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving rdh12 |
| MY204271A (en) | 2017-11-15 | 2024-08-20 | Ucl Business Ltd | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies |
| TR2021022284A2 (tr) | 2021-12-31 | 2022-01-21 | T C Ueskuedar Ueniversitesi | Retinitis pigmentosa (rp) hastalığının tedavisinde kullanılmak üzere retinol dehidrojenaz 12 (rdh12) gen mutasyonlarına yönelik crıspr-pe sistemi. |
-
2017
- 2017-07-07 WO PCT/US2017/041122 patent/WO2018009814A1/en not_active Ceased
- 2017-07-07 JP JP2019500531A patent/JP7101656B2/ja active Active
- 2017-07-07 KR KR1020197003500A patent/KR102526506B1/ko active Active
- 2017-07-07 IL IL297095A patent/IL297095B2/en unknown
- 2017-07-07 BR BR112019000171A patent/BR112019000171A2/pt not_active Application Discontinuation
- 2017-07-07 AU AU2017292010A patent/AU2017292010B2/en active Active
- 2017-07-07 RU RU2019103488A patent/RU2764920C2/ru active
- 2017-07-07 US US16/314,179 patent/US11197936B2/en active Active
- 2017-07-07 EP EP17824989.2A patent/EP3481958A4/en active Pending
- 2017-07-07 IL IL264070A patent/IL264070B2/en unknown
- 2017-07-07 CN CN201780054628.1A patent/CN110191954B/zh active Active
- 2017-07-07 KR KR1020237013817A patent/KR102799528B1/ko active Active
- 2017-07-07 IL IL320533A patent/IL320533A/en unknown
- 2017-07-07 CA CA3029646A patent/CA3029646A1/en active Pending
-
2021
- 2021-11-10 US US17/523,492 patent/US20230037283A9/en active Pending
-
2022
- 2022-01-06 US US17/569,896 patent/US12419973B2/en active Active
- 2022-05-06 JP JP2022076693A patent/JP7572990B2/ja active Active
- 2022-08-26 US US17/822,533 patent/US20230009257A1/en active Pending
-
2023
- 2023-10-17 AU AU2023251411A patent/AU2023251411A1/en active Pending
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