JP2019511216A5 - - Google Patents
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- Publication number
- JP2019511216A5 JP2019511216A5 JP2018545818A JP2018545818A JP2019511216A5 JP 2019511216 A5 JP2019511216 A5 JP 2019511216A5 JP 2018545818 A JP2018545818 A JP 2018545818A JP 2018545818 A JP2018545818 A JP 2018545818A JP 2019511216 A5 JP2019511216 A5 JP 2019511216A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- rna
- seq
- vector
- synthetic
- 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
- 229920002477 rna polymer Polymers 0.000 claims 23
- 239000013598 vector Substances 0.000 claims 10
- 239000004055 small Interfering RNA Substances 0.000 claims 9
- 108091027967 Small hairpin RNA Proteins 0.000 claims 7
- 101150079354 rho gene Proteins 0.000 claims 7
- 108700011259 MicroRNAs Proteins 0.000 claims 6
- 230000002452 interceptive effect Effects 0.000 claims 6
- 239000002679 microRNA Substances 0.000 claims 6
- 230000009452 underexpressoin Effects 0.000 claims 5
- 108091081021 Sense strand Proteins 0.000 claims 4
- 230000000692 anti-sense effect Effects 0.000 claims 4
- 230000003252 repetitive effect Effects 0.000 claims 3
- 239000013603 viral vector Substances 0.000 claims 3
- 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 2
- 241000124008 Mammalia Species 0.000 claims 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 2
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 2
- 108091026821 Artificial microRNA Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 102000009572 RNA Polymerase II Human genes 0.000 claims 1
- 108010009460 RNA Polymerase II Proteins 0.000 claims 1
- 102000014450 RNA Polymerase III Human genes 0.000 claims 1
- 108010078067 RNA Polymerase III Proteins 0.000 claims 1
- 241000283984 Rodentia Species 0.000 claims 1
- 239000002671 adjuvant Substances 0.000 claims 1
- 239000000969 carrier Substances 0.000 claims 1
- 239000013613 expression plasmid Substances 0.000 claims 1
- 230000014509 gene expression Effects 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 229940035893 uracil Drugs 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022156496A JP7642211B2 (ja) | 2016-03-01 | 2022-09-29 | 優性網膜色素変性の治療のためのａａｖベクター |
| JP2024168632A JP2025016451A (ja) | 2016-03-01 | 2024-09-27 | 優性網膜色素変性の治療のためのａａｖベクター |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662302122P | 2016-03-01 | 2016-03-01 | |
| US62/302,122 | 2016-03-01 | ||
| US201662398451P | 2016-09-22 | 2016-09-22 | |
| US62/398,451 | 2016-09-22 | ||
| PCT/US2017/020289 WO2017151823A1 (en) | 2016-03-01 | 2017-03-01 | Aav vectors for treatment of dominant retinitis pigmentosa |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022156496A Division JP7642211B2 (ja) | 2016-03-01 | 2022-09-29 | 優性網膜色素変性の治療のためのａａｖベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019511216A JP2019511216A (ja) | 2019-04-25 |
| JP2019511216A5 true JP2019511216A5 (cg-RX-API-DMAC7.html) | 2020-04-09 |
| JP7272795B2 JP7272795B2 (ja) | 2023-05-12 |
Family
ID=59743213
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018545818A Active JP7272795B2 (ja) | 2016-03-01 | 2017-03-01 | 優性網膜色素変性の治療のためのａａｖベクター |
| JP2022156496A Active JP7642211B2 (ja) | 2016-03-01 | 2022-09-29 | 優性網膜色素変性の治療のためのａａｖベクター |
| JP2024168632A Pending JP2025016451A (ja) | 2016-03-01 | 2024-09-27 | 優性網膜色素変性の治療のためのａａｖベクター |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022156496A Active JP7642211B2 (ja) | 2016-03-01 | 2022-09-29 | 優性網膜色素変性の治療のためのａａｖベクター |
| JP2024168632A Pending JP2025016451A (ja) | 2016-03-01 | 2024-09-27 | 優性網膜色素変性の治療のためのａａｖベクター |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11118185B2 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3423582B1 (cg-RX-API-DMAC7.html) |
| JP (3) | JP7272795B2 (cg-RX-API-DMAC7.html) |
| CN (2) | CN109154002B (cg-RX-API-DMAC7.html) |
| AU (2) | AU2017227776C1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3014671A1 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2923877T3 (cg-RX-API-DMAC7.html) |
| IL (2) | IL261014B2 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017151823A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109154002B (zh) | 2016-03-01 | 2022-08-30 | 佛罗里达大学研究基金会有限公司 | 用于治疗显性视网膜色素变性的aav载体 |
| US20230203508A1 (en) * | 2017-11-07 | 2023-06-29 | The University Of North Carolina At Chapel Hill | Methods and compositions for circular rna molecules |
| SG11202011151VA (en) * | 2018-05-15 | 2020-12-30 | Univ Washington | Compositions and methods for reducing spliceopathy and treating rna dominance disorders |
| WO2019232517A1 (en) * | 2018-06-01 | 2019-12-05 | University Of Florida Research Foundation, Incorporated | Compositions and methods for treatment of dominant retinitis pigmentosa |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| CN111518813B (zh) * | 2019-02-03 | 2023-04-28 | 武汉纽福斯生物科技有限公司 | 视紫红质的编码序列、其表达载体构建及其应用 |
| US20220288101A1 (en) * | 2019-08-15 | 2022-09-15 | The Children's Hospital Of Philadelphia | Combined transgene and intron-derived mirna therapy for treatment of sca1 |
| CN113038971B (zh) * | 2020-04-21 | 2022-06-24 | 北京中因科技有限公司 | RHO-adRP基于基因编辑的方法和组合物 |
| CN111926044B (zh) * | 2020-10-12 | 2021-01-22 | 北京大学第三医院（北京大学第三临床医学院） | 结合突变rho基因的核酸分子和试剂盒 |
| JP2024505257A (ja) | 2021-02-01 | 2024-02-05 | レジェンクスバイオ インコーポレーテッド | 神経セロイドリポフスチン症の遺伝子治療 |
| WO2025231487A1 (en) * | 2024-05-03 | 2025-11-06 | The Trustees Of The University Of Pennsylvania | Photoreceptor specific promotors for use in gene therapies for treatment of retinal degeneration |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| GB9606961D0 (en) | 1996-04-02 | 1996-06-05 | Farrar Gwyneth J | Genetic strategy III |
| US20040234999A1 (en) | 1996-04-02 | 2004-11-25 | Farrar Gwenyth Jane | Genetic suppression and replacement |
| US8551970B2 (en) | 1996-04-02 | 2013-10-08 | Optigen Patents Limited | Genetic suppression and replacement |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| WO2004094606A2 (en) | 2003-04-18 | 2004-11-04 | The Trustees Of The University Of Pennsylvania | COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF ANGIOPOIETIN 1 AND 2 AND THEIR RECEPTOR TIE2 |
| US20070009899A1 (en) * | 2003-10-02 | 2007-01-11 | Mounts William M | Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases |
| EP1735442A2 (en) | 2004-03-24 | 2006-12-27 | Oncotherapy Science, Inc. | Compositions and methods for treating pancreatic cancer |
| US20080221057A1 (en) | 2007-02-16 | 2008-09-11 | Wyeth | Secreted protein ccdc80 regulates adipocyte differentiation |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| EP2140004B1 (en) * | 2007-04-12 | 2022-01-12 | The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin | Genetic suppression and replacement |
| US20110129825A1 (en) * | 2007-08-03 | 2011-06-02 | Melba Stinnett Ketchum | Compositions, methods and systems for the simultaneous determination of parentage, identity, sex, genotype and/or phenotype and breed determination in animals |
| WO2010127209A2 (en) * | 2009-04-30 | 2010-11-04 | The Research Foundation Of State University Of New York | Compositions and methods for therapy of macular degeneration |
| EP2449113B8 (en) | 2010-07-30 | 2015-11-25 | CureVac AG | Complexation of nucleic acids with disulfide-crosslinked cationic components for transfection and immunostimulation |
| CN102061308B (zh) | 2010-10-29 | 2013-05-08 | 北京未名凯拓作物设计中心有限公司 | 一种用百草枯筛选转基因植物的方法 |
| CN102061305B (zh) | 2010-12-15 | 2012-07-18 | 深圳市百恩维生物科技有限公司 | 一种多ShRNA高效协同沉默基因方法及载体 |
| US20130064815A1 (en) | 2011-09-12 | 2013-03-14 | The Trustees Of Princeton University | Inducing apoptosis in quiescent cells |
| US9695443B2 (en) * | 2012-05-25 | 2017-07-04 | Commissariat A L'energie Atomique Et Aux Energies Alternatives | Vector for the selective silencing of a gene in astrocytes |
| WO2014138792A1 (en) | 2013-03-14 | 2014-09-18 | Commonwealth Scientific And Industrial Research Organisation | Double-stranded rna |
| PT3119437T (pt) * | 2014-03-21 | 2019-12-12 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
| ES2848377T3 (es) | 2015-02-26 | 2021-08-09 | Ionis Pharmaceuticals Inc | Moduladores específicos de alelo de RODOPSINA P23H |
| DK3289080T3 (da) | 2015-04-30 | 2021-11-08 | Univ Columbia | Genterapi til autosomalt dominante sygdomme |
| WO2017137493A1 (en) | 2016-02-09 | 2017-08-17 | Fondazione Telethon | Synthetic promoters and uses thereof |
| US20170348387A1 (en) | 2016-02-29 | 2017-12-07 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for nphp5 lca-ciliopathy |
| CN109154002B (zh) | 2016-03-01 | 2022-08-30 | 佛罗里达大学研究基金会有限公司 | 用于治疗显性视网膜色素变性的aav载体 |
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2017
- 2017-03-01 CN CN201780014309.8A patent/CN109154002B/zh active Active
- 2017-03-01 JP JP2018545818A patent/JP7272795B2/ja active Active
- 2017-03-01 EP EP17760752.0A patent/EP3423582B1/en active Active
- 2017-03-01 US US16/081,307 patent/US11118185B2/en active Active
- 2017-03-01 CA CA3014671A patent/CA3014671A1/en active Pending
- 2017-03-01 WO PCT/US2017/020289 patent/WO2017151823A1/en not_active Ceased
- 2017-03-01 EP EP22170988.4A patent/EP4119668A1/en active Pending
- 2017-03-01 AU AU2017227776A patent/AU2017227776C1/en active Active
- 2017-03-01 IL IL261014A patent/IL261014B2/en unknown
- 2017-03-01 CN CN202210934990.0A patent/CN115896106A/zh active Pending
- 2017-03-01 IL IL302455A patent/IL302455A/en unknown
- 2017-03-01 ES ES17760752T patent/ES2923877T3/es active Active
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2021
- 2021-05-21 US US17/327,609 patent/US20210324387A1/en active Pending
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2022
- 2022-09-29 JP JP2022156496A patent/JP7642211B2/ja active Active
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2023
- 2023-09-27 AU AU2023237106A patent/AU2023237106B2/en active Active
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2024
- 2024-09-27 JP JP2024168632A patent/JP2025016451A/ja active Pending