JP2015501156A5 - - Google Patents
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- JP2015501156A5 JP2015501156A5 JP2014539038A JP2014539038A JP2015501156A5 JP 2015501156 A5 JP2015501156 A5 JP 2015501156A5 JP 2014539038 A JP2014539038 A JP 2014539038A JP 2014539038 A JP2014539038 A JP 2014539038A JP 2015501156 A5 JP2015501156 A5 JP 2015501156A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- cells
- composition
- viral vector
- rdcvf
- 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.)
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- 108020004707 nucleic acids Proteins 0.000 claims description 75
- 102000039446 nucleic acids Human genes 0.000 claims description 75
- 150000007523 nucleic acids Chemical class 0.000 claims description 75
- 210000004027 cell Anatomy 0.000 claims description 68
- 101000597433 Mus musculus Nucleoredoxin-like protein 1 Proteins 0.000 claims description 48
- 239000013603 viral vector Substances 0.000 claims description 44
- 239000002773 nucleotide Substances 0.000 claims description 38
- 125000003729 nucleotide group Chemical group 0.000 claims description 38
- 238000000034 method Methods 0.000 claims description 30
- 239000007924 injection Substances 0.000 claims description 16
- 238000002347 injection Methods 0.000 claims description 16
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 15
- 201000010099 disease Diseases 0.000 claims description 13
- 108091026890 Coding region Proteins 0.000 claims description 12
- 241000124008 Mammalia Species 0.000 claims description 12
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 10
- 102000004169 proteins and genes Human genes 0.000 claims description 7
- 108090000623 proteins and genes Proteins 0.000 claims description 7
- 239000013598 vector Substances 0.000 claims description 7
- 206010003694 Atrophy Diseases 0.000 claims description 6
- 241000701022 Cytomegalovirus Species 0.000 claims description 6
- 150000001413 amino acids Chemical group 0.000 claims description 6
- 230000037444 atrophy Effects 0.000 claims description 6
- 208000011325 dry age related macular degeneration Diseases 0.000 claims description 6
- 208000011580 syndromic disease Diseases 0.000 claims description 6
- 239000000825 pharmaceutical preparation Substances 0.000 claims description 5
- 230000028327 secretion Effects 0.000 claims description 5
- 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 description 4
- 239000013607 AAV vector Substances 0.000 claims description 4
- 102100026031 Beta-glucuronidase Human genes 0.000 claims description 4
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 claims description 4
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims description 4
- 108020004705 Codon Proteins 0.000 claims description 4
- 101000599951 Homo sapiens Insulin-like growth factor I Proteins 0.000 claims description 4
- 102000002265 Human Growth Hormone Human genes 0.000 claims description 4
- 108010000521 Human Growth Hormone Proteins 0.000 claims description 4
- 239000000854 Human Growth Hormone Substances 0.000 claims description 4
- 102100037852 Insulin-like growth factor I Human genes 0.000 claims description 4
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 4
- 229940077737 brain-derived neurotrophic factor Drugs 0.000 claims description 4
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
- 208000024827 Alzheimer disease Diseases 0.000 claims description 2
- 208000023328 Basedow disease Diseases 0.000 claims description 2
- 241000713704 Bovine immunodeficiency virus Species 0.000 claims description 2
- 208000002177 Cataract Diseases 0.000 claims description 2
- 206010007747 Cataract congenital Diseases 0.000 claims description 2
- 108020004414 DNA Proteins 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 206010012689 Diabetic retinopathy Diseases 0.000 claims description 2
- 208000003098 Ganglion Cysts Diseases 0.000 claims description 2
- 208000010412 Glaucoma Diseases 0.000 claims description 2
- 108010060309 Glucuronidase Proteins 0.000 claims description 2
- 208000015023 Graves' disease Diseases 0.000 claims description 2
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 2
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 2
- 101000933465 Homo sapiens Beta-glucuronidase Proteins 0.000 claims description 2
- 101000597428 Homo sapiens Nucleoredoxin-like protein 1 Proteins 0.000 claims description 2
- 208000023105 Huntington disease Diseases 0.000 claims description 2
- 208000032578 Inherited retinal disease Diseases 0.000 claims description 2
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 claims description 2
- 102100036205 Nucleoredoxin-like protein 2 Human genes 0.000 claims description 2
- 101710106007 Nucleoredoxin-like protein 2 Proteins 0.000 claims description 2
- 206010030043 Ocular hypertension Diseases 0.000 claims description 2
- 208000018737 Parkinson disease Diseases 0.000 claims description 2
- 208000032430 Retinal dystrophy Diseases 0.000 claims description 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 2
- 208000027073 Stargardt disease Diseases 0.000 claims description 2
- 208000005400 Synovial Cyst Diseases 0.000 claims description 2
- 208000014769 Usher Syndromes Diseases 0.000 claims description 2
- 206010046851 Uveitis Diseases 0.000 claims description 2
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims description 2
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 2
- 210000002159 anterior chamber Anatomy 0.000 claims description 2
- 238000004113 cell culture Methods 0.000 claims description 2
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims description 2
- 239000012228 culture supernatant Substances 0.000 claims description 2
- 238000012258 culturing Methods 0.000 claims description 2
- 208000035475 disorder Diseases 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 208000030533 eye disease Diseases 0.000 claims description 2
- 201000006321 fundus dystrophy Diseases 0.000 claims description 2
- 210000002287 horizontal cell Anatomy 0.000 claims description 2
- 102000057445 human NXNL1 Human genes 0.000 claims description 2
- 210000005260 human cell Anatomy 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims description 2
- 238000001727 in vivo Methods 0.000 claims description 2
- 208000017532 inherited retinal dystrophy Diseases 0.000 claims description 2
- 230000002401 inhibitory effect Effects 0.000 claims description 2
- 208000029515 lens disease Diseases 0.000 claims description 2
- 210000004962 mammalian cell Anatomy 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 229930182817 methionine Natural products 0.000 claims description 2
- 238000000746 purification Methods 0.000 claims description 2
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 claims description 2
- 210000001685 thyroid gland Anatomy 0.000 claims description 2
- 210000003501 vero cell Anatomy 0.000 claims description 2
- 238000009736 wetting Methods 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 29
- 230000003376 axonal effect Effects 0.000 claims 1
- 230000008786 sensory perception of smell Effects 0.000 claims 1
- 241000700605 Viruses Species 0.000 description 1
- 210000003050 axon Anatomy 0.000 description 1
- 230000000977 initiatory effect Effects 0.000 description 1
- 239000008194 pharmaceutical composition Substances 0.000 description 1
- 230000003248 secreting effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161552155P | 2011-10-27 | 2011-10-27 | |
| US61/552,155 | 2011-10-27 | ||
| PCT/US2012/062106 WO2013063383A2 (en) | 2011-10-27 | 2012-10-26 | Vectors encoding rod-derived cone viability factor |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015501156A JP2015501156A (ja) | 2015-01-15 |
| JP2015501156A5 true JP2015501156A5 (enExample) | 2015-12-17 |
| JP6293664B2 JP6293664B2 (ja) | 2018-03-14 |
Family
ID=48168788
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014539038A Expired - Fee Related JP6293664B2 (ja) | 2011-10-27 | 2012-10-26 | 桿体由来錐体生存因子をコードするベクター |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US9265813B2 (enExample) |
| EP (1) | EP2797613B1 (enExample) |
| JP (1) | JP6293664B2 (enExample) |
| KR (1) | KR102000141B1 (enExample) |
| CN (2) | CN104321069A (enExample) |
| AU (1) | AU2012321102C1 (enExample) |
| BR (1) | BR112014010091B1 (enExample) |
| CA (1) | CA2853379C (enExample) |
| DK (1) | DK2797613T3 (enExample) |
| ES (1) | ES2774779T3 (enExample) |
| MX (1) | MX354516B (enExample) |
| PL (1) | PL2797613T3 (enExample) |
| RU (1) | RU2664673C2 (enExample) |
| WO (1) | WO2013063383A2 (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2027889A1 (en) * | 2007-06-05 | 2009-02-25 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | New neuronal viability factor and use thereof |
| ES2796737T3 (es) * | 2012-10-17 | 2020-11-30 | Inst Nat Sante Rech Med | Métodos y composiciones farmacéuticas para el tratamiento de la degeneración macular relacionada con la edad (AMD) |
| DK3546585T3 (da) * | 2014-04-25 | 2025-11-03 | Genethon | Nukleinsyrer med en modificeret intron til brug i behandlingen af hyperbilirubinæmi |
| WO2016185242A1 (en) * | 2015-05-21 | 2016-11-24 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Synergistic combination of neuronal viability factors and uses thereof |
| GB201516066D0 (en) | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
| JP7061067B2 (ja) | 2015-12-14 | 2022-04-27 | 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 | クリグラー・ナジャー症候群の処置のための組成物 |
| WO2017120294A1 (en) | 2016-01-05 | 2017-07-13 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of ocular disorders and blinding diseases |
| CN106148528B (zh) * | 2016-07-11 | 2019-10-01 | 赵晨 | 一种遗传性Usher综合征的致病突变及其检测试剂 |
| CA3025977A1 (en) * | 2016-10-11 | 2018-04-19 | Wellstat Ophthalmics Corporation | Fusion protein between short form rod-derived cone viability factor and a hydrophilic peptide |
| EP3697896A1 (en) * | 2017-10-16 | 2020-08-26 | Vigeneron GmbH | Aav vectors |
| SG11202006796RA (en) * | 2017-12-22 | 2020-08-28 | Sparingvision | Constructs comprising neuronal viability factors and uses thereof |
| CA3088239A1 (en) * | 2018-01-30 | 2019-08-08 | Universiteit Gent | Compositions for use to treat cataract |
| CN108504685A (zh) * | 2018-03-27 | 2018-09-07 | 宜明细胞生物科技有限公司 | 一种利用CRISPR/Cas9系统同源重组修复IL-2RG缺陷基因的方法 |
| SG11202102140WA (en) * | 2018-09-07 | 2021-04-29 | Univ Southern California | Cryopreservation of cell-seeded substrates and related methods |
| SG11202104295UA (en) * | 2018-11-14 | 2021-06-29 | Regenxbio Inc | Gene therapy for neuronal ceroid lipofuscinoses |
| CN113454227B (zh) * | 2018-12-19 | 2025-05-23 | 维萨梅布有限公司 | 编码蛋白质的rna |
| EP4028413A1 (en) | 2019-09-10 | 2022-07-20 | Obsidian Therapeutics, Inc. | Ca2-il15 fusion proteins for tunable regulation |
| EP4074828B1 (en) * | 2019-12-09 | 2026-03-04 | Chigenovo Co., Ltd. | Uses of cyp4v2 and rdcvf for use in treating bietti's crystalline dystrophy |
| CN111733174B (zh) * | 2020-08-07 | 2021-02-09 | 北京大学第三医院（北京大学第三临床医学院） | 一种分离的核酸分子及其用途 |
| JP7811576B2 (ja) * | 2020-08-28 | 2026-02-05 | ジョイント・ストック・カンパニー “バイオキャド” | ＳＡＲＳ－ＣｏＶ－２に対するＡＡＶ５に基づくワクチン |
| KR20260008735A (ko) | 2023-03-30 | 2026-01-16 | 파마 싱크, 엘엘씨 | 간상체-유래 원뿔세포 생존인자 및 인간 IgK 신호 서열을 인코딩하는 벡터 |
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| US5840293A (en) | 1988-11-16 | 1998-11-24 | Advanced Polymer Systems, Inc. | Ionic beads for controlled release and adsorption |
| US5225347A (en) | 1989-09-25 | 1993-07-06 | Innovir Laboratories, Inc. | Therapeutic ribozyme compositions and expression vectors |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
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| HK1001832A1 (en) | 1991-04-25 | 1998-07-10 | Brown University Research Foundation | Implantable biocompatible immunoisolatory vehicle for delivery of selected therapeutic products |
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| AU5013893A (en) | 1992-08-14 | 1994-03-15 | Rockefeller University, The | Herpesviral defective vector with rat preproenkephalin gene promoter |
| WO1994008598A1 (en) | 1992-10-09 | 1994-04-28 | Advanced Tissue Sciences, Inc. | Liver reserve cells |
| GB9223084D0 (en) | 1992-11-04 | 1992-12-16 | Imp Cancer Res Tech | Compounds to target cells |
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| DE19541450C2 (de) | 1995-11-07 | 1997-10-02 | Gsf Forschungszentrum Umwelt | Genkonstrukt und dessen Verwendung |
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| JP2005533485A (ja) | 2002-02-04 | 2005-11-10 | ノバルティス アクチエンゲゼルシャフト | 組換えウシ免疫不全ウイルスに基づく遺伝子導入システム |
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| FR2870241B1 (fr) | 2004-05-13 | 2015-02-27 | Novartis Ag | Facteur de viabilite des cones derive des batonnets ou rdcvf et applications |
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| WO2009134681A2 (en) | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
| JP5748223B2 (ja) | 2008-09-10 | 2015-07-15 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | ニューロン生存因子およびその使用 |
| CN102272302B (zh) * | 2008-10-31 | 2015-04-15 | 生物载体株式会社 | 增强重组蛋白表达的方法 |
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-
2012
- 2012-10-26 MX MX2014004949A patent/MX354516B/es active IP Right Grant
- 2012-10-26 CA CA2853379A patent/CA2853379C/en active Active
- 2012-10-26 WO PCT/US2012/062106 patent/WO2013063383A2/en not_active Ceased
- 2012-10-26 RU RU2014121256A patent/RU2664673C2/ru active
- 2012-10-26 CN CN201280064474.1A patent/CN104321069A/zh active Pending
- 2012-10-26 KR KR1020147011857A patent/KR102000141B1/ko active Active
- 2012-10-26 CN CN201911125836.3A patent/CN110777124A/zh active Pending
- 2012-10-26 AU AU2012321102A patent/AU2012321102C1/en active Active
- 2012-10-26 ES ES12843778T patent/ES2774779T3/es active Active
- 2012-10-26 PL PL12843778T patent/PL2797613T3/pl unknown
- 2012-10-26 US US14/354,415 patent/US9265813B2/en active Active
- 2012-10-26 BR BR112014010091-8A patent/BR112014010091B1/pt active IP Right Grant
- 2012-10-26 EP EP12843778.7A patent/EP2797613B1/en active Active
- 2012-10-26 DK DK12843778.7T patent/DK2797613T3/da active
- 2012-10-26 JP JP2014539038A patent/JP6293664B2/ja not_active Expired - Fee Related
-
2015
- 2015-12-08 US US14/962,337 patent/US10040835B2/en active Active
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