JP2016535034A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
- Authority
- JP
- Japan
- Prior art keywords
- vector
- composition
- administration
- nucleic acid
- acid molecule
- 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
- 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 (enExample) | 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 (enExample) |
| EP (3) | EP4331590A3 (enExample) |
| JP (3) | JP6317440B2 (enExample) |
| WO (1) | WO2015066190A1 (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10980896B2 (en) | 2013-10-29 | 2021-04-20 | President And Fellows Of Harvard College | Methods and compositions for inhibiting oxidative stress |
| 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 |
| 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 |
| 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 |
| 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 |
| US20190049465A1 (en) * | 2016-02-06 | 2019-02-14 | 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 |
| BR112020005436B1 (pt) | 2017-09-20 | 2022-08-02 | 4D Molecular Therapeutics Inc | Proteína do capsídeo de variante do vírus adenoassociado, virion do aav recombinante (raav) infeccioso, composições, composições farmacêuticas e usos de virion de raav ou de composições farmacêuticas |
| AU2019249162A1 (en) * | 2018-04-03 | 2020-11-26 | Cornell University | Gene therapy for oxidative stress |
| MX2021002060A (es) | 2018-08-20 | 2021-07-21 | Janssen Pharmaceutica Nv | Inhibidores de la interacción proteína-proteína 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 |
| US20220088222A1 (en) * | 2018-12-20 | 2022-03-24 | President And Fellows Of Harvard College | Compositions and methods for the treatment of degenerative ocular diseases |
| US20220118107A1 (en) * | 2019-02-11 | 2022-04-21 | President And Fellows Of Harvard College | Txnip and ldhb compositions and methods for the treatment of degenerative ocular diseases |
| KR20230026986A (ko) | 2020-04-27 | 2023-02-27 | 4디 몰레큘러 테라퓨틱스 아이엔씨. | 코돈 최적화된 gla 유전자 및 이의 용도 |
| WO2021226037A1 (en) * | 2020-05-04 | 2021-11-11 | Sulfagenix, Inc. | Treatment of viral infections |
| WO2021250081A1 (en) * | 2020-06-09 | 2021-12-16 | Genethon | Nrf2 activator for use in treating dilated cardiomyopathies |
| 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 |
Family Cites Families (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US4373071A (en) | 1981-04-30 | 1983-02-08 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4401796A (en) | 1981-04-30 | 1983-08-30 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| US4598049A (en) | 1983-08-31 | 1986-07-01 | Systec Inc. | General purpose gene synthesizer |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US5116742A (en) | 1986-12-03 | 1992-05-26 | University Patents, Inc. | RNA ribozyme restriction endoribonucleases and methods |
| US4987071A (en) | 1986-12-03 | 1991-01-22 | University Patents, Inc. | RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods |
| US5716826A (en) | 1988-03-21 | 1998-02-10 | Chiron Viagene, Inc. | Recombinant retroviruses |
| US5591624A (en) | 1988-03-21 | 1997-01-07 | Chiron Viagene, Inc. | Retroviral packaging cell lines |
| CA1336396C (en) | 1988-07-25 | 1995-07-25 | Kiyoshi Kita | Intraocular anticoagulant including antithrombin iii and method of administration |
| US5223409A (en) | 1988-09-02 | 1993-06-29 | Protein Engineering Corp. | Directed evolution of novel binding proteins |
| US5175384A (en) | 1988-12-05 | 1992-12-29 | Genpharm International | Transgenic mice depleted in mature t-cells and methods for making transgenic mice |
| US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| ES2087997T3 (es) | 1990-01-12 | 1996-08-01 | Cell Genesys Inc | Generacion de anticuerpos xenogenicos. |
| US6075181A (en) | 1990-01-12 | 2000-06-13 | Abgenix, Inc. | Human antibodies derived from immunized xenomice |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| US5427908A (en) | 1990-05-01 | 1995-06-27 | Affymax Technologies N.V. | Recombinant library screening methods |
| JPH06508511A (ja) | 1990-07-10 | 1994-09-29 | ケンブリッジ アンティボディー テクノロジー リミティド | 特異的な結合ペアーの構成員の製造方法 |
| GB9015198D0 (en) | 1990-07-10 | 1990-08-29 | Brien Caroline J O | Binding substance |
| DE69129154T2 (de) | 1990-12-03 | 1998-08-20 | Genentech, Inc., South San Francisco, Calif. | Verfahren zur anreicherung von proteinvarianten mit geänderten bindungseigenschaften |
| DE69233769D1 (de) | 1991-03-01 | 2009-09-24 | Dyax Corp | Chimäres Protein mit Mikroprotein mit zwei oder mehr Disulfidbindungen und Ausgestaltungen davon |
| ATE414768T1 (de) | 1991-04-10 | 2008-12-15 | Scripps Research Inst | Bibliotheken heterodimerer rezeptoren mittels phagemiden |
| DE4122599C2 (de) | 1991-07-08 | 1993-11-11 | Deutsches Krebsforsch | Phagemid zum Screenen von Antikörpern |
| MX9204374A (es) | 1991-07-25 | 1993-03-01 | Idec Pharma Corp | Anticuerpo recombinante y metodo para su produccion. |
| US5756096A (en) | 1991-07-25 | 1998-05-26 | Idec Pharmaceuticals Corporation | Recombinant antibodies for human therapy |
| JPH06509578A (ja) | 1991-07-26 | 1994-10-27 | ユニバーシティ・オブ・ロチェスター | 悪性細胞利用による癌治療法 |
| US5529774A (en) | 1991-08-13 | 1996-06-25 | The Regents Of The University Of California | In vivo transfer of the HSV-TK gene implanted retroviral producer cells |
| JP3571337B2 (ja) | 1992-02-11 | 2004-09-29 | セル ジェネシス，インコーポレーテッド | 遺伝子標的現象による同型遺伝子接合 |
| US5631236A (en) | 1993-08-26 | 1997-05-20 | Baylor College Of Medicine | Gene therapy for solid tumors, using a DNA sequence encoding HSV-Tk or VZV-Tk |
| CA2169557A1 (en) | 1993-09-03 | 1995-03-09 | Edward H. Oldfield | Treatment of human tumors by genetic transformation of human tumor cells |
| US5763217A (en) | 1993-11-10 | 1998-06-09 | University Of British Columbia | Method of using, process of preparing and composition comprising recombinant herpesvirus vectors |
| US5714353A (en) | 1994-05-24 | 1998-02-03 | Research Corporation Technologies, Inc. | Safe vectors for gene therapy |
| US5688773A (en) | 1994-08-17 | 1997-11-18 | The General Hospital Corporation | Method of selectively destroying neoplastic cells |
| US5763242A (en) | 1995-02-08 | 1998-06-09 | Thomas Jefferson University | Method for increasing transduction efficiency of recombinant retroviral vectors |
| US5756283A (en) | 1995-06-05 | 1998-05-26 | The Trustees Of The University Of Pennsylvania | Method for improved production of recombinant adeno-associated viruses for gene therapy |
| US5985846A (en) | 1995-06-07 | 1999-11-16 | Baylor College Of Medicine | Gene therapy for muscular dystrophy |
| US5811524A (en) | 1995-06-07 | 1998-09-22 | Idec Pharmaceuticals Corporation | Neutralizing high affinity human monoclonal antibodies specific to RSV F-protein and methods for their manufacture and therapeutic use thereof |
| US5994132A (en) | 1996-10-23 | 1999-11-30 | University Of Michigan | Adenovirus vectors |
| US5981225A (en) | 1998-04-16 | 1999-11-09 | Baylor College Of Medicine | Gene transfer vector, recombinant adenovirus particles containing the same, method for producing the same and method of use of the same |
| AU3629200A (en) | 1999-03-15 | 2000-10-04 | John Guy | Recombinant adeno-associated virus transfer of genes affecting demyelination |
| US20020049176A1 (en) * | 1999-11-10 | 2002-04-25 | Anderson Christen M. | Modulation of mitochondrial mass and function for the treatment of diseases and for target and drug discovery |
| JP2004514407A (ja) | 2000-04-28 | 2004-05-20 | 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 | 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター |
| WO2005003766A2 (en) | 2003-06-13 | 2005-01-13 | Whitehead Institute For Biomedical Research | Methods of regulating metabolism and mitochondrial function |
| US20080274093A1 (en) * | 2004-12-02 | 2008-11-06 | Johnson Jeffrey A | Method of diminishing the symptoms of neurodegenerative disease |
| WO2007005879A2 (en) * | 2005-07-01 | 2007-01-11 | The Johns Hopkins University | Compositions and methods for the treatment or prevention of disorders relating to oxidative stress |
| AU2007324073A1 (en) | 2006-10-23 | 2008-05-29 | John Guy | Suppression of mitochondrial oxidative stress |
| EP2680006A1 (en) | 2007-02-08 | 2014-01-01 | Biogen Idec MA Inc. | Treatment for Amyotrophic Lateral Sclerosis |
| US20120108654A1 (en) * | 2008-06-30 | 2012-05-03 | The Johns Hopkins University | Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa |
| MX2011009751A (es) * | 2009-03-16 | 2011-09-29 | Opko Curna Llc | Tratamiento de enfermedades relacionadas con factor nuclear (eritroide derivado 2) tipo 2 (nrf2) por inhibicion del transcrito antisentido natural a nrf2. |
| US9610363B2 (en) | 2009-04-16 | 2017-04-04 | President And Fellows Of Harvard College | Methods for inhibiting starvation of a cell |
| US20130288985A1 (en) * | 2010-07-15 | 2013-10-31 | The Schepens Eye Research Institute Inc. | Compositions and methods of treatment of corneal endothelium disorders |
| US10980896B2 (en) | 2013-10-29 | 2021-04-20 | President And Fellows Of Harvard College | Methods and compositions for inhibiting oxidative stress |
| US11879133B2 (en) * | 2017-04-24 | 2024-01-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
-
2014
- 2014-10-29 US US15/032,426 patent/US10980896B2/en active Active
- 2014-10-29 EP EP23213942.8A patent/EP4331590A3/en active Pending
- 2014-10-29 WO PCT/US2014/062917 patent/WO2015066190A1/en not_active Ceased
- 2014-10-29 EP EP14857455.1A patent/EP3062884B1/en active Active
- 2014-10-29 JP JP2016526853A patent/JP6317440B2/ja active Active
- 2014-10-29 EP EP20196298.2A patent/EP3777980B1/en active Active
-
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
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2016535034A5 (enExample) | ||
| Liu et al. | Gene therapy for ocular diseases | |
| Colella et al. | Ocular gene therapy: current progress and future prospects | |
| Ramlogan‐Steel et al. | Gene therapy and the adeno‐associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: trials, future directions and safety considerations | |
| Zhou et al. | Activation of the extracellular signal-regulated kinase 1/2 pathway by AAV gene transfer protects retinal ganglion cells in glaucoma | |
| Yu-Wai-Man | Genetic manipulation for inherited neurodegenerative diseases: myth or reality? | |
| Bennett et al. | Gene therapy for ocular disease | |
| JP2019205465A (ja) | 錐体細胞における増強された遺伝子発現のための組成物および方法 | |
| JP2018062532A5 (enExample) | ||
| Ratican et al. | Progress in gene therapy to prevent retinal ganglion cell loss in glaucoma and Leber’s hereditary optic neuropathy | |
| CA2986630C (en) | Synergistic combination of neuronal viability factors and uses thereof | |
| HRP20211024T4 (hr) | Aav vektori za gensku terapiju mrežnice i cns-a | |
| JP2018529336A (ja) | 網膜色素変性症の治療 | |
| Gargiulo et al. | AAV-mediated tyrosinase gene transfer restores melanogenesis and retinal function in a model of oculo-cutaneous albinism type I (OCA1) | |
| Sundaresan et al. | Therapeutic applications of CRISPR/Cas9 gene editing technology for the treatment of ocular diseases | |
| Dalkara et al. | Gene therapy for the eye focus on mutation-independent approaches | |
| JP2024059871A (ja) | コドン最適化補体因子ｉ | |
| Wu et al. | Retinal degeneration: molecular mechanisms and therapeutic strategies | |
| JP7211960B2 (ja) | 眼疾患の遺伝子治療 | |
| Petit et al. | Gene therapy approaches for the treatment of retinal disorders | |
| Hojo et al. | Photoreceptor protection by iris pigment epithelial transplantation transduced with AAV-mediated brain-derived neurotrophic factor gene | |
| Chekuri et al. | AAV2-mediated intravitreal delivery of exon-specific U1 snRNA rescues optic neuropathy in familial dysautonomia | |
| Raz-Prag et al. | Photoreceptor Protection by Adeno-Associated Virus–Mediated LEDGF Expression in the RCS Rat Model of Retinal Degeneration: Probing the Mechanism | |
| Colella et al. | AAV-mediated gene supply for treatment of degenerative and neovascular retinal diseases | |
| Petersen‐Jones | Viral vectors for targeting the canine retina: a review |