JP2022106918A5 - - Google Patents
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- Publication number
- JP2022106918A5 JP2022106918A5 JP2022076693A JP2022076693A JP2022106918A5 JP 2022106918 A5 JP2022106918 A5 JP 2022106918A5 JP 2022076693 A JP2022076693 A JP 2022076693A JP 2022076693 A JP2022076693 A JP 2022076693A JP 2022106918 A5 JP2022106918 A5 JP 2022106918A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- rdh12
- human
- dna
- use according
- 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
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- 101000742938 Homo sapiens Retinol dehydrogenase 12 Proteins 0.000 claims 8
- 102000046986 human RDH12 Human genes 0.000 claims 8
- 101710178616 Retinol dehydrogenase 12 Proteins 0.000 claims 4
- 102100038054 Retinol dehydrogenase 12 Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 239000013607 AAV vector Substances 0.000 claims 3
- 102000004437 G-Protein-Coupled Receptor Kinase 1 Human genes 0.000 claims 3
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 3
- 208000022873 Ocular disease Diseases 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 108091004242 G-Protein-Coupled Receptor Kinase 1 Proteins 0.000 claims 2
- 201000010480 Leber congenital amaurosis 13 Diseases 0.000 claims 2
- 108090000799 Rhodopsin kinases Proteins 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 101000611338 Homo sapiens Rhodopsin Proteins 0.000 claims 1
- 101150033365 Rdh12 gene Proteins 0.000 claims 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 208000030533 eye disease Diseases 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 230000004777 loss-of-function mutation Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 239000002245 particle Substances 0.000 claims 1
- 108091008695 photoreceptors Proteins 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662359777P | 2016-07-08 | 2016-07-08 | |
| US62/359,777 | 2016-07-08 | ||
| JP2019500531A 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 |
| PCT/US2017/041122 WO2018009814A1 (en) | 2016-07-08 | 2017-07-07 | Methods and compositions for treatment of disorders and diseases involving rdh12 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019500531A Division 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022106918A JP2022106918A (ja) | 2022-07-20 |
| JP2022106918A5 true JP2022106918A5 (enExample) | 2022-12-06 |
| JP7572990B2 JP7572990B2 (ja) | 2024-10-24 |
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 Before (1)
| 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 |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US11197936B2 (enExample) |
| EP (1) | EP3481958B1 (enExample) |
| JP (2) | JP7101656B2 (enExample) |
| KR (2) | KR102799528B1 (enExample) |
| CN (1) | CN110191954B (enExample) |
| AU (2) | AU2017292010B2 (enExample) |
| BR (1) | BR112019000171A2 (enExample) |
| CA (1) | CA3029646A1 (enExample) |
| IL (3) | IL320533A (enExample) |
| RU (1) | RU2764920C2 (enExample) |
| WO (1) | WO2018009814A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3359663A4 (en) * | 2015-09-24 | 2019-08-14 | The Trustees of The University of Pennsylvania | COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE |
| KR102799528B1 (ko) | 2016-07-08 | 2025-04-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물 |
| 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 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | 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)
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|---|---|---|---|---|
| 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 |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| AU728220B2 (en) | 1997-04-14 | 2001-01-04 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| JP2001517454A (ja) | 1997-09-19 | 2001-10-09 | 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 | 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株 |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| 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 |
| CA2410901C (en) | 2000-06-01 | 2012-03-20 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| WO2002082904A2 (en) | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| AU2003301148A1 (en) | 2002-12-23 | 2004-07-22 | Vical Incorporated | Codon-optimized polynucleotide-based vaccines against human cytomegalovirus infection |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| 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 |
| EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| WO2006132118A1 (ja) | 2005-06-09 | 2006-12-14 | Matsushita Electric Industrial Co., Ltd. | 振幅誤差補償装置及び直交度誤差補償装置 |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| WO2009105690A2 (en) | 2008-02-21 | 2009-08-27 | Targeted Genetics Corporation | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| US7561972B1 (en) | 2008-06-06 | 2009-07-14 | Dna Twopointo, Inc. | Synthetic nucleic acids for expression of encoded proteins |
| 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 |
| 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 |
| KR20130040844A (ko) | 2010-03-29 | 2013-04-24 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 약학적으로 유발된 전이유전자 제거 시스템 |
| KR20200047642A (ko) * | 2010-04-23 | 2020-05-07 | 유니버시티 오브 플로리다 리서치 파운데이션, 인크. | 레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법 |
| 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 |
| EP2872183B1 (en) | 2012-07-11 | 2018-09-26 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
| US8962449B1 (en) | 2013-07-30 | 2015-02-24 | Micron Technology, Inc. | Methods for processing semiconductor devices |
| WO2015075154A2 (en) * | 2013-11-20 | 2015-05-28 | Fondazione Telethon | Artificial dna-binding proteins and uses thereof |
| EP3800191B1 (en) | 2014-03-17 | 2025-08-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| EP3137497B2 (en) * | 2014-05-02 | 2025-03-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| US10392622B2 (en) * | 2014-08-01 | 2019-08-27 | 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 |
| KR102799528B1 (ko) | 2016-07-08 | 2025-04-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물 |
| 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 KR KR1020237013817A patent/KR102799528B1/ko active Active
- 2017-07-07 AU AU2017292010A patent/AU2017292010B2/en active Active
- 2017-07-07 CN CN201780054628.1A patent/CN110191954B/zh active Active
- 2017-07-07 BR BR112019000171A patent/BR112019000171A2/pt not_active Application Discontinuation
- 2017-07-07 WO PCT/US2017/041122 patent/WO2018009814A1/en not_active Ceased
- 2017-07-07 EP EP17824989.2A patent/EP3481958B1/en active Active
- 2017-07-07 US US16/314,179 patent/US11197936B2/en active Active
- 2017-07-07 IL IL320533A patent/IL320533A/en unknown
- 2017-07-07 KR KR1020197003500A patent/KR102526506B1/ko active Active
- 2017-07-07 JP JP2019500531A patent/JP7101656B2/ja active Active
- 2017-07-07 RU RU2019103488A patent/RU2764920C2/ru active
- 2017-07-07 IL IL264070A patent/IL264070B2/en unknown
- 2017-07-07 IL IL297095A patent/IL297095B2/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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