JP2020512822A5 - - Google Patents
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- Publication number
- JP2020512822A5 JP2020512822A5 JP2019555010A JP2019555010A JP2020512822A5 JP 2020512822 A5 JP2020512822 A5 JP 2020512822A5 JP 2019555010 A JP2019555010 A JP 2019555010A JP 2019555010 A JP2019555010 A JP 2019555010A JP 2020512822 A5 JP2020512822 A5 JP 2020512822A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- isolated nucleic
- seq
- composition
- cep290
- 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
- 108020004707 nucleic acids Proteins 0.000 claims 21
- 102000039446 nucleic acids Human genes 0.000 claims 21
- 150000007523 nucleic acids Chemical class 0.000 claims 21
- 239000000203 mixture Substances 0.000 claims 15
- 102100035673 Centrosomal protein of 290 kDa Human genes 0.000 claims 10
- 101710198317 Centrosomal protein of 290 kDa Proteins 0.000 claims 10
- 239000012634 fragment Substances 0.000 claims 9
- 150000001413 amino acids Chemical group 0.000 claims 7
- 230000035772 mutation Effects 0.000 claims 7
- 108700019146 Transgenes Proteins 0.000 claims 6
- 108090000565 Capsid Proteins Proteins 0.000 claims 5
- 102100023321 Ceruloplasmin Human genes 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 108091008695 photoreceptors Proteins 0.000 claims 4
- 101150078156 Cep290 gene Proteins 0.000 claims 3
- 230000001886 ciliary effect Effects 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 201000004569 Blindness Diseases 0.000 claims 2
- 206010010356 Congenital anomaly Diseases 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
- 102100023742 Rhodopsin kinase GRK1 Human genes 0.000 claims 2
- 230000037430 deletion Effects 0.000 claims 2
- 238000012217 deletion Methods 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 208000011580 syndromic disease Diseases 0.000 claims 2
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical compound O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 claims 1
- 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
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 208000026372 Congenital cystic kidney disease Diseases 0.000 claims 1
- 108091004242 G-Protein-Coupled Receptor Kinase 1 Proteins 0.000 claims 1
- 101000829506 Homo sapiens Rhodopsin kinase GRK1 Proteins 0.000 claims 1
- 201000008645 Joubert syndrome Diseases 0.000 claims 1
- 201000008643 Meckel syndrome Diseases 0.000 claims 1
- 108020004485 Nonsense Codon Proteins 0.000 claims 1
- 201000007737 Retinal degeneration Diseases 0.000 claims 1
- 102000004330 Rhodopsin Human genes 0.000 claims 1
- 108090000820 Rhodopsin Proteins 0.000 claims 1
- 108090000799 Rhodopsin kinases Proteins 0.000 claims 1
- 208000014769 Usher Syndromes Diseases 0.000 claims 1
- 208000027418 Wounds and injury Diseases 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 206010001902 amaurosis Diseases 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 210000004081 cilia Anatomy 0.000 claims 1
- 230000006378 damage Effects 0.000 claims 1
- 230000007850 degeneration Effects 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 231100000221 frame shift mutation induction Toxicity 0.000 claims 1
- 230000037433 frameshift Effects 0.000 claims 1
- 208000014674 injury Diseases 0.000 claims 1
- 208000018769 loss of vision Diseases 0.000 claims 1
- 231100000864 loss of vision Toxicity 0.000 claims 1
- 201000002648 nephronophthisis Diseases 0.000 claims 1
- 230000037434 nonsense mutation Effects 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 230000004258 retinal degeneration Effects 0.000 claims 1
- 230000004283 retinal dysfunction Effects 0.000 claims 1
- 102000029752 retinol binding Human genes 0.000 claims 1
- 108091000053 retinol binding Proteins 0.000 claims 1
- 238000011200 topical administration Methods 0.000 claims 1
- 230000004393 visual impairment Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023020149A JP7535332B2 (ja) | 2017-04-05 | 2023-02-13 | ミニ遺伝子療法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762481727P | 2017-04-05 | 2017-04-05 | |
| US62/481,727 | 2017-04-05 | ||
| PCT/US2018/026230 WO2018187552A1 (en) | 2017-04-05 | 2018-04-05 | Minigene therapy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023020149A Division JP7535332B2 (ja) | 2017-04-05 | 2023-02-13 | ミニ遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020512822A JP2020512822A (ja) | 2020-04-30 |
| JP2020512822A5 true JP2020512822A5 (enExample) | 2021-05-13 |
| JP7307480B2 JP7307480B2 (ja) | 2023-07-12 |
Family
ID=63712901
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019555010A Active JP7307480B2 (ja) | 2017-04-05 | 2018-04-05 | ミニ遺伝子療法 |
| JP2023020149A Active JP7535332B2 (ja) | 2017-04-05 | 2023-02-13 | ミニ遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023020149A Active JP7535332B2 (ja) | 2017-04-05 | 2023-02-13 | ミニ遺伝子療法 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US11739346B2 (enExample) |
| EP (1) | EP3606544B1 (enExample) |
| JP (2) | JP7307480B2 (enExample) |
| CN (2) | CN118360335A (enExample) |
| AU (2) | AU2018248304C1 (enExample) |
| CA (1) | CA3059112A1 (enExample) |
| WO (1) | WO2018187552A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2925049T3 (es) * | 2015-11-16 | 2022-10-13 | Synlogic Operating Co Inc | Bacterias manipuladas para reducir la hiperfenilalaninemia |
| CN118360335A (zh) | 2017-04-05 | 2024-07-19 | 马萨诸塞大学 | 小基因治疗 |
| WO2020214796A1 (en) * | 2019-04-19 | 2020-10-22 | University Of Massachusetts | Gene therapies for usher syndrome (ush2a) |
| WO2020214797A1 (en) | 2019-04-19 | 2020-10-22 | University Of Massachusetts | Gene therapies for usher syndrome (ush1b) |
| WO2020236815A1 (en) * | 2019-05-20 | 2020-11-26 | University Of Massachusetts | Minigene therapy |
| GB2700048B (en) | 2019-09-18 | 2026-03-04 | Aldevron Llc | synthetic dna vectors and method of use |
| EP4189098A1 (en) | 2020-07-27 | 2023-06-07 | Anjarium Biosciences AG | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| CN112062827B (zh) * | 2020-09-16 | 2022-10-21 | 中国人民解放军军事科学院军事医学研究院 | Cep55蛋白在调控纤毛去组装和制备纤毛相关疾病模型中的应用 |
| US11674154B2 (en) * | 2020-12-03 | 2023-06-13 | University Of Massachusetts | Gene therapeutics for fibrodysplasia ossificans progressiva |
| WO2022169774A1 (en) * | 2021-02-02 | 2022-08-11 | University Of Massachusetts | Inverted terminal repeats from aav serotypes 8 and rh.39 in gene therapy vectors |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5549910A (en) | 1989-03-31 | 1996-08-27 | The Regents Of The University Of California | Preparation of liposome and lipid complex compositions |
| JP3218637B2 (ja) | 1990-07-26 | 2001-10-15 | 大正製薬株式会社 | 安定なリポソーム水懸濁液 |
| JP2958076B2 (ja) | 1990-08-27 | 1999-10-06 | 株式会社ビタミン研究所 | 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法 |
| US5399363A (en) | 1991-01-25 | 1995-03-21 | Eastman Kodak Company | Surface modified anticancer nanoparticles |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| US5741516A (en) | 1994-06-20 | 1998-04-21 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| US5738868A (en) | 1995-07-18 | 1998-04-14 | Lipogenics Ltd. | Liposome compositions and kits therefor |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 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 |
| EP2262899A1 (en) | 2008-04-02 | 2010-12-22 | Fondazione Telethon | Method of treating genetic disorders |
| US10266845B2 (en) | 2013-02-08 | 2019-04-23 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| ES2704677T3 (es) | 2013-04-18 | 2019-03-19 | Fond Telethon | Suministro eficaz de genes grandes por vectores de AAV duales |
| WO2015009575A1 (en) | 2013-07-16 | 2015-01-22 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of disorders related to cep290 |
| EP3030665B9 (en) * | 2013-08-05 | 2023-10-04 | Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum | Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp |
| ES2745769T3 (es) | 2014-03-10 | 2020-03-03 | Editas Medicine Inc | Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10) |
| WO2016033338A1 (en) * | 2014-08-27 | 2016-03-03 | The Usa, As Represented By The Secretary, Dept. Of Health And Human Services | Methods and compositions for treating leber congenital amaurosis |
| WO2017087900A1 (en) | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| JP7007273B2 (ja) | 2015-12-22 | 2022-01-24 | アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） | 遺伝子治療用の改良された複合型二重組換えａａｖベクターシステム |
| US20170348387A1 (en) * | 2016-02-29 | 2017-12-07 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for nphp5 lca-ciliopathy |
| CA3032822A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| CN118360335A (zh) | 2017-04-05 | 2024-07-19 | 马萨诸塞大学 | 小基因治疗 |
| BR112019019015A2 (pt) | 2017-06-30 | 2020-04-14 | Univ California | vírions de vírus adeno-associado com capsídeos variantes e seus métodos de uso |
| US11723988B2 (en) | 2017-10-20 | 2023-08-15 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector |
| WO2020236815A1 (en) | 2019-05-20 | 2020-11-26 | University Of Massachusetts | Minigene therapy |
-
2018
- 2018-04-05 CN CN202410492766.XA patent/CN118360335A/zh active Pending
- 2018-04-05 JP JP2019555010A patent/JP7307480B2/ja active Active
- 2018-04-05 CA CA3059112A patent/CA3059112A1/en active Pending
- 2018-04-05 US US16/500,238 patent/US11739346B2/en active Active
- 2018-04-05 CN CN201880037056.0A patent/CN110709095B/zh active Active
- 2018-04-05 AU AU2018248304A patent/AU2018248304C1/en active Active
- 2018-04-05 WO PCT/US2018/026230 patent/WO2018187552A1/en not_active Ceased
- 2018-04-05 EP EP18781286.2A patent/EP3606544B1/en active Active
-
2023
- 2023-02-13 JP JP2023020149A patent/JP7535332B2/ja active Active
- 2023-02-16 AU AU2023200880A patent/AU2023200880B2/en active Active
- 2023-06-30 US US18/345,366 patent/US12454702B2/en active Active
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