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 (https=) | 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 (https=) |
| EP (1) | EP3606544B1 (https=) |
| JP (2) | JP7307480B2 (https=) |
| CN (2) | CN110709095B (https=) |
| AU (2) | AU2018248304C1 (https=) |
| CA (1) | CA3059112A1 (https=) |
| WO (1) | WO2018187552A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017087580A1 (en) * | 2015-11-16 | 2017-05-26 | Synlogic, Inc. | Bacteria engineered to reduce hyperphenylalaninemia |
| US11739346B2 (en) | 2017-04-05 | 2023-08-29 | University Of Massachusetts | Minigene therapy |
| EP3956453A4 (en) | 2019-04-19 | 2023-02-22 | University of Massachusetts | GENE THERAPIES FOR USHER SYNDROME (USH1B) |
| CN114402075A (zh) * | 2019-04-19 | 2022-04-26 | 马萨诸塞大学 | 乌谢尔综合征(ush2a)的基因疗法 |
| CN114206353B (zh) * | 2019-05-20 | 2024-11-08 | 马萨诸塞大学 | 小基因疗法 |
| GB2700048B (en) | 2019-09-18 | 2026-03-04 | Aldevron Llc | synthetic dna vectors and method of use |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| 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 |
| EP4255457A4 (en) * | 2020-12-03 | 2025-05-21 | University of Massachusetts | DEVELOPMENT OF NEW GENE THERAPEUTIC AGENTS FOR FIBRODYSPLASIA OSSIFICANS PROGRESSIVA |
| US11926842B2 (en) * | 2021-02-02 | 2024-03-12 | University Of Massachusetts | Use of inverted terminal repeats (ITRS) from adeno-associated virus 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 |
| AU722624B2 (en) | 1996-09-06 | 2000-08-10 | Trustees Of The University Of Pennsylvania, The | 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 |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| US20110117058A1 (en) | 2008-04-02 | 2011-05-19 | 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 |
| CA2909733C (en) | 2013-04-18 | 2023-10-17 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| 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 |
| WO2015138510A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 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 |
| EP3377116A4 (en) | 2015-11-19 | 2019-07-10 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR CORRECTING ECG-RELATED EYE DISEASE |
| WO2017108931A1 (en) | 2015-12-22 | 2017-06-29 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Improved hybrid dual recombinant aav vector systems for gene therapy |
| US20170348387A1 (en) * | 2016-02-29 | 2017-12-07 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for nphp5 lca-ciliopathy |
| WO2018026976A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| US11739346B2 (en) | 2017-04-05 | 2023-08-29 | University Of Massachusetts | Minigene therapy |
| WO2019006182A1 (en) | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF |
| JP7420710B2 (ja) | 2017-10-20 | 2024-01-23 | 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） | 組換えａａｖ９由来ベクターの治療有効量の網膜下送達を含む、対象の錐体視細胞において目的のポリヌクレオチドを発現させる方法 |
| CN114206353B (zh) | 2019-05-20 | 2024-11-08 | 马萨诸塞大学 | 小基因疗法 |
-
2018
- 2018-04-05 US US16/500,238 patent/US11739346B2/en active Active
- 2018-04-05 AU AU2018248304A patent/AU2018248304C1/en active Active
- 2018-04-05 CN CN201880037056.0A patent/CN110709095B/zh active Active
- 2018-04-05 CN CN202410492766.XA patent/CN118360335A/zh active Pending
- 2018-04-05 WO PCT/US2018/026230 patent/WO2018187552A1/en not_active Ceased
- 2018-04-05 EP EP18781286.2A patent/EP3606544B1/en active Active
- 2018-04-05 CA CA3059112A patent/CA3059112A1/en active Pending
- 2018-04-05 JP JP2019555010A patent/JP7307480B2/ja 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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