JP2016535034A5 - - Google Patents
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- JP2016535034A5 JP2016535034A5 JP2016526853A JP2016526853A JP2016535034A5 JP 2016535034 A5 JP2016535034 A5 JP 2016535034A5 JP 2016526853 A JP2016526853 A JP 2016526853A JP 2016526853 A JP2016526853 A JP 2016526853A JP 2016535034 A5 JP2016535034 A5 JP 2016535034A5
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- vector
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- nucleic acid
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- 239000013598 vector Substances 0.000 claims description 74
- 239000000203 mixture Substances 0.000 claims description 62
- 102000039446 nucleic acids Human genes 0.000 claims description 36
- 108020004707 nucleic acids Proteins 0.000 claims description 36
- 150000007523 nucleic acids Chemical class 0.000 claims description 36
- 210000004027 cell Anatomy 0.000 claims description 26
- 101000588302 Homo sapiens Nuclear factor erythroid 2-related factor 2 Proteins 0.000 claims description 20
- 102100031701 Nuclear factor erythroid 2-related factor 2 Human genes 0.000 claims description 20
- 230000006851 antioxidant defense Effects 0.000 claims description 16
- 108090000623 proteins and genes Proteins 0.000 claims description 16
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 15
- 239000013607 AAV vector Substances 0.000 claims description 12
- 230000036542 oxidative stress Effects 0.000 claims description 12
- 230000002207 retinal effect Effects 0.000 claims description 12
- 210000000608 photoreceptor cell Anatomy 0.000 claims description 11
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 10
- 102000004169 proteins and genes Human genes 0.000 claims description 10
- 208000030533 eye disease Diseases 0.000 claims description 9
- 239000008194 pharmaceutical composition Substances 0.000 claims description 9
- 102100032891 Superoxide dismutase [Mn], mitochondrial Human genes 0.000 claims description 8
- 239000003795 chemical substances by application Substances 0.000 claims description 8
- 230000000694 effects Effects 0.000 claims description 8
- 230000002401 inhibitory effect Effects 0.000 claims description 8
- 108010045815 superoxide dismutase 2 Proteins 0.000 claims description 8
- 241000701161 unidentified adenovirus Species 0.000 claims description 8
- 241001430294 unidentified retrovirus Species 0.000 claims description 8
- 102000016938 Catalase Human genes 0.000 claims description 6
- 108010053835 Catalase Proteins 0.000 claims description 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 6
- 208000035475 disorder Diseases 0.000 claims description 6
- 230000001771 impaired effect Effects 0.000 claims description 6
- 239000013603 viral vector Substances 0.000 claims description 6
- 208000022873 Ocular disease Diseases 0.000 claims description 5
- 206010057430 Retinal injury Diseases 0.000 claims description 5
- 241000702421 Dependoparvovirus Species 0.000 claims description 4
- 241000991587 Enterovirus C Species 0.000 claims description 4
- 241000701074 Human alphaherpesvirus 2 Species 0.000 claims description 4
- 241000713666 Lentivirus Species 0.000 claims description 4
- 241001631646 Papillomaviridae Species 0.000 claims description 4
- 241000125945 Protoparvovirus Species 0.000 claims description 4
- 241000712909 Reticuloendotheliosis virus Species 0.000 claims description 4
- 241000700584 Simplexvirus Species 0.000 claims description 4
- 241000700618 Vaccinia virus Species 0.000 claims description 4
- 230000003833 cell viability Effects 0.000 claims description 4
- 201000006754 cone-rod dystrophy Diseases 0.000 claims description 4
- 239000003642 reactive oxygen metabolite Substances 0.000 claims description 4
- 210000001525 retina Anatomy 0.000 claims description 3
- 230000004083 survival effect Effects 0.000 claims description 3
- 208000036443 AIPL1-related retinopathy Diseases 0.000 claims description 2
- 108090000790 Enzymes Proteins 0.000 claims description 2
- 102000004190 Enzymes Human genes 0.000 claims description 2
- 208000026350 Inborn Genetic disease Diseases 0.000 claims description 2
- 102000003921 Peroxisome Proliferator-Activated Receptor Gamma Coactivator 1-alpha Human genes 0.000 claims description 2
- 108090000310 Peroxisome Proliferator-Activated Receptor Gamma Coactivator 1-alpha Proteins 0.000 claims description 2
- 208000017442 Retinal disease Diseases 0.000 claims description 2
- 102000040945 Transcription factor Human genes 0.000 claims description 2
- 108091023040 Transcription factor Proteins 0.000 claims description 2
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 2
- 239000003963 antioxidant agent Substances 0.000 claims description 2
- 230000003078 antioxidant effect Effects 0.000 claims description 2
- 230000007850 degeneration Effects 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 229940079593 drug Drugs 0.000 claims description 2
- 210000003743 erythrocyte Anatomy 0.000 claims description 2
- 208000016361 genetic disease Diseases 0.000 claims description 2
- 208000002780 macular degeneration Diseases 0.000 claims description 2
- 239000000049 pigment Substances 0.000 claims description 2
- 230000002792 vascular Effects 0.000 claims description 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 230000035899 viability Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 41
- 210000003370 receptor cell Anatomy 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361896805P | 2013-10-29 | 2013-10-29 | |
| US61/896,805 | 2013-10-29 | ||
| PCT/US2014/062917 WO2015066190A1 (en) | 2013-10-29 | 2014-10-29 | Methods and compositions for inhibting oxidative stress |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018010718A Division JP6440875B2 (ja) | 2013-10-29 | 2018-01-25 | 酸化ストレスを阻害するための方法および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016535034A JP2016535034A (ja) | 2016-11-10 |
| JP2016535034A5 true JP2016535034A5 (cg-RX-API-DMAC10.html) | 2017-06-22 |
| JP6317440B2 JP6317440B2 (ja) | 2018-04-25 |
Family
ID=53005074
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016526853A Active JP6317440B2 (ja) | 2013-10-29 | 2014-10-29 | 酸化ストレスを阻害するための方法および組成物 |
| JP2018010718A Active JP6440875B2 (ja) | 2013-10-29 | 2018-01-25 | 酸化ストレスを阻害するための方法および組成物 |
| JP2018217337A Pending JP2019048858A (ja) | 2013-10-29 | 2018-11-20 | 酸化ストレスを阻害するための方法および組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018010718A Active JP6440875B2 (ja) | 2013-10-29 | 2018-01-25 | 酸化ストレスを阻害するための方法および組成物 |
| JP2018217337A Pending JP2019048858A (ja) | 2013-10-29 | 2018-11-20 | 酸化ストレスを阻害するための方法および組成物 |
Country Status (4)
| Country | Link |
|---|---|
| US (3) | US10980896B2 (cg-RX-API-DMAC10.html) |
| EP (3) | EP3062884B1 (cg-RX-API-DMAC10.html) |
| JP (3) | JP6317440B2 (cg-RX-API-DMAC10.html) |
| WO (1) | WO2015066190A1 (cg-RX-API-DMAC10.html) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6317440B2 (ja) | 2013-10-29 | 2018-04-25 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 酸化ストレスを阻害するための方法および組成物 |
| RU2651757C2 (ru) * | 2016-01-19 | 2018-04-23 | Селл энд Джин Терапи Лтд | Линейка биологически активных генно-терапевтических субстанций на основе гена sod2 для коррекции патологических состояний клеток органов и тканей и органов и тканей человека, связанных с оксидативным стрессом, способ получения и использования |
| RU2651758C2 (ru) * | 2016-01-20 | 2018-04-23 | Селл энд Джин Терапи Лтд | Средство для коррекции патологических состояний клеток органов и тканей и/или органов и тканей человека, на основе гена gpx3, связанных с оксидативным стрессом, способ получения и использования |
| RU2653491C2 (ru) * | 2016-01-20 | 2018-05-08 | Селл энд Джин Терапи Лтд | Линейка биологически активных генно-терапевтических субстанций на основе гена gpx1 для коррекции патологических состояний клеток органов и тканей и органов и тканей человека, связанных с оксидативным стрессом, способ получения и использования |
| RU2649820C2 (ru) * | 2016-01-20 | 2018-04-04 | Селл энд Джин Терапи Лтд | Средство для коррекции патологических состояний клеток органов и тканей и/или органов и тканей человека на основе гена col1a2, связанных с количественным снижением белка альфа-2 цепи коллагена i типа |
| WO2017136805A1 (en) * | 2016-02-06 | 2017-08-10 | Georgetown University | Compositions and methods for the diagnosis and treatment of age-related macular degeneration |
| RU2651048C1 (ru) * | 2016-11-21 | 2018-04-18 | Общество с ограниченной ответственностью "Аллель Центр Инновационных Биотехнологий" | Средство для лечения состояний человеческого организма, связанных с уменьшением уровня экспрессии гена il-11 и/или уменьшением количества белка интерлейкина-11 на основе генно-терапевтических субстанций с геном il-11, способ получения и использования |
| RU2653487C1 (ru) * | 2016-11-30 | 2018-05-08 | Общество с ограниченной ответственностью "Аллель Центр Инновационных Биотехнологий" | Средство для лечения состояний человеческого организма, связанных с уменьшением уровня экспрессии гена lif и/или уменьшением количества белка фактора, ингибирующего лейкемию, на основе генно-терапевтических субстанций с геном lif, способ получения и использования |
| RU2649814C1 (ru) * | 2017-02-14 | 2018-04-04 | Общество с ограниченной ответственностью "Медсервис" | Средство для лечения состояний человеческого организма, связанных с уменьшением уровня экспрессии гена сат и/или уменьшением активности белка каталазы на основе генно-терапевтических субстанций с геном сат, способ получения и использования |
| JP7105532B2 (ja) * | 2017-05-10 | 2022-07-25 | ロート製薬株式会社 | 後眼部疾患の予防、改善、又は治療用組成物 |
| DK3684423T5 (da) | 2017-09-20 | 2024-08-26 | 4D Molecular Therapeutics Inc | Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf |
| US20210381002A1 (en) * | 2018-04-03 | 2021-12-09 | Cornell University | Gene therapy for oxidative stress |
| JOP20210030A1 (ar) | 2018-08-20 | 2021-02-16 | Janssen Pharmaceutica Nv | مثبطات تفاعل البروتين-بروتين KEAP1-Nrf2 |
| CN109055428A (zh) * | 2018-09-19 | 2018-12-21 | 上海市第人民医院 | 一种重组腺相关病毒载体及其制备方法与应用 |
| WO2020132040A1 (en) * | 2018-12-20 | 2020-06-25 | President And Fellows Of Harvard College | Compositions and methods for the treatment of degenerative ocular diseases |
| WO2020167770A1 (en) * | 2019-02-11 | 2020-08-20 | President And Fellows Of Harvard College | Txnip and ldhb compositions and methods for the treatment of degenerative ocular diseases |
| CA3175905A1 (en) | 2020-04-27 | 2021-11-04 | David H. Kirn | Codon optimized gla genes and uses thereof |
| CA3177580A1 (en) * | 2020-05-04 | 2021-11-11 | Sulfagenix, Inc. | Treatment of viral infections |
| JP2023528661A (ja) * | 2020-06-09 | 2023-07-05 | ジェネトン | 拡張型心筋症の処置における使用のためのｎｒｆ２活性化剤 |
| KR102620997B1 (ko) * | 2022-11-10 | 2024-01-04 | 주식회사 아이두젠 | 노각나무 추출물을 유효성분으로 포함하는 시력 보호용 또는 망막 질환의 개선 및 예방용 조성물 |
| WO2025110266A1 (ko) * | 2023-11-21 | 2025-05-30 | 주식회사 퓨어바이오 | 시력 보호 또는 망막 질환의 개선, 예방 또는 치료를 위한 노각나무 추출물의 용도 |
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| WO2010005533A2 (en) * | 2008-06-30 | 2010-01-14 | The Johns Hopkins University | Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa |
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| US20130288985A1 (en) * | 2010-07-15 | 2013-10-31 | The Schepens Eye Research Institute Inc. | Compositions and methods of treatment of corneal endothelium disorders |
| JP6317440B2 (ja) | 2013-10-29 | 2018-04-25 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 酸化ストレスを阻害するための方法および組成物 |
| US11879133B2 (en) * | 2017-04-24 | 2024-01-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
-
2014
- 2014-10-29 JP JP2016526853A patent/JP6317440B2/ja active Active
- 2014-10-29 EP EP14857455.1A patent/EP3062884B1/en active Active
- 2014-10-29 EP EP20196298.2A patent/EP3777980B1/en active Active
- 2014-10-29 US US15/032,426 patent/US10980896B2/en active Active
- 2014-10-29 WO PCT/US2014/062917 patent/WO2015066190A1/en not_active Ceased
- 2014-10-29 EP EP23213942.8A patent/EP4331590A3/en active Pending
-
2018
- 2018-01-25 JP JP2018010718A patent/JP6440875B2/ja active Active
- 2018-11-20 JP JP2018217337A patent/JP2019048858A/ja active Pending
-
2021
- 2021-03-19 US US17/206,212 patent/US12042546B2/en active Active
-
2024
- 2024-04-19 US US18/640,031 patent/US20240350673A1/en active Pending
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