JP2022106918A5 - - Google Patents
Download PDFInfo
- 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
Links
- 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 (12)
| 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) |
| ES (1) | ES3069917T3 (enExample) |
| IL (3) | IL264070B2 (enExample) |
| RU (1) | RU2764920C2 (enExample) |
| WO (1) | WO2018009814A1 (enExample) |
Families Citing this family (10)
| 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 |
| US11197936B2 (en) * | 2016-07-08 | 2021-12-14 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving RDH12 |
| CA3099004C (en) | 2017-05-04 | 2024-11-12 | Lehigh University | ADDITIVE MANUFACTURING SYSTEM WITH ADJUSTABLE MATERIAL PROPERTIES |
| SMT202400321T1 (it) * | 2017-11-15 | 2024-11-15 | Univ Michigan Regents | Vettori virali comprendenti regioni codificanti rdh12 e metodi di trattamento di distrofie retiniche |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| EP4457347A4 (en) * | 2021-12-31 | 2026-07-01 | T C Uskudar Univ | CRISPR-PE SYSTEM FOR RETINOL DEHYDROGENASE 12 (RDH12) GENE MUTATIONS FOR THE TREATMENT OF RETINITIS PIGMENTARIAN (RP) |
| 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 |
| EP1007637B1 (en) | 1997-04-14 | 2004-06-30 | 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 |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| CA2442670A1 (en) | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| WO2004058166A2 (en) | 2002-12-23 | 2004-07-15 | 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 |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| 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 |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| 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 |
| HUE041571T2 (hu) * | 2010-04-23 | 2019-05-28 | Univ Florida | RAAV-guanilát-cikláz-készítmények és ejárások 1-es típusú Leber-féle veleszületett vakság (LCA1) kezelésére |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| WO2013151664A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| CN105120901A (zh) | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
| 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 | Universita Degli Studi Di Napoli Federico Ii | Artificial DNA-Binding Proteins and Their Uses |
| ES2822551T3 (es) * | 2014-03-17 | 2021-05-04 | Adverum Biotechnologies Inc | Composiciones y métodos para la expresión génica mejorada en células de los conos |
| LT3137497T (lt) | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| 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 |
| US11197936B2 (en) | 2016-07-08 | 2021-12-14 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving RDH12 |
| SMT202400321T1 (it) | 2017-11-15 | 2024-11-15 | Univ Michigan Regents | Vettori virali comprendenti regioni codificanti rdh12 e metodi di trattamento di distrofie retiniche |
| 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 US US16/314,179 patent/US11197936B2/en active Active
- 2017-07-07 KR KR1020237013817A patent/KR102799528B1/ko active Active
- 2017-07-07 JP JP2019500531A patent/JP7101656B2/ja active Active
- 2017-07-07 WO PCT/US2017/041122 patent/WO2018009814A1/en not_active Ceased
- 2017-07-07 AU AU2017292010A patent/AU2017292010B2/en active Active
- 2017-07-07 ES ES17824989T patent/ES3069917T3/es active Active
- 2017-07-07 KR KR1020197003500A patent/KR102526506B1/ko active Active
- 2017-07-07 IL IL264070A patent/IL264070B2/en unknown
- 2017-07-07 IL IL320533A patent/IL320533A/en unknown
- 2017-07-07 RU RU2019103488A patent/RU2764920C2/ru active
- 2017-07-07 EP EP17824989.2A patent/EP3481958B1/en active Active
- 2017-07-07 CA CA3029646A patent/CA3029646A1/en active Pending
- 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 IL IL297095A patent/IL297095B2/en unknown
-
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/AU2023251411B2/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2026027271A5 (enExample) | ||
| US12281309B1 (en) | Composition for regulating production of interfering ribonucleic acid | |
| JP2019524090A5 (enExample) | ||
| US12522847B2 (en) | Plasmid encoding an insulin peptide and Fc fusion protein | |
| US12264329B1 (en) | Composition for regulating production of fusion proteins | |
| US12522832B2 (en) | Composition for regulating production of interfering ribonucleic acid | |
| US12480121B1 (en) | Composition for regulating production of interfering ribonucleic acid | |
| US12480129B1 (en) | Composition for regulating production of interfering ribonucleic acid | |
| US12584129B1 (en) | Composition for regulating production of interfering ribonucleic acid | |
| US12529061B1 (en) | Composition for regulating production of interfering ribonucleic acid | |
| US12486510B2 (en) | Composition for regulating production of interfering ribonucleic acid | |
| US12529060B2 (en) | Composition for regulating production of interfering ribonucleic acid | |
| WO2025065890A1 (zh) | 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用 | |
| Goya et al. | Neuroendocrinology of aging: the potential of gene therapy as an interventive strategy |