JP2017512466A5 - - Google Patents
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- JP2017512466A5 JP2017512466A5 JP2016556768A JP2016556768A JP2017512466A5 JP 2017512466 A5 JP2017512466 A5 JP 2017512466A5 JP 2016556768 A JP2016556768 A JP 2016556768A JP 2016556768 A JP2016556768 A JP 2016556768A JP 2017512466 A5 JP2017512466 A5 JP 2017512466A5
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- Prior art keywords
- nucleic acid
- hotc
- sequence
- seq
- acid sequence
- 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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- 239000013603 viral vector Substances 0.000 claims 16
- 150000007523 nucleic acids Chemical group 0.000 claims 14
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 13
- 239000013598 vector Substances 0.000 claims 10
- 102100028200 Ornithine transcarbamylase, mitochondrial Human genes 0.000 claims 8
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 claims 6
- 241000700605 Viruses Species 0.000 claims 5
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 108091026890 Coding region Proteins 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 206010016654 Fibrosis Diseases 0.000 claims 2
- 101000986595 Homo sapiens Ornithine transcarbamylase, mitochondrial Proteins 0.000 claims 2
- 102100027105 Lymphocyte-specific protein 1 Human genes 0.000 claims 2
- 208000000599 Ornithine Carbamoyltransferase Deficiency Disease Diseases 0.000 claims 2
- 206010052450 Ornithine transcarbamoylase deficiency Diseases 0.000 claims 2
- 208000035903 Ornithine transcarbamylase deficiency Diseases 0.000 claims 2
- 102000002248 Thyroxine-Binding Globulin Human genes 0.000 claims 2
- 108010000259 Thyroxine-Binding Globulin Proteins 0.000 claims 2
- 239000004480 active ingredient Substances 0.000 claims 2
- 210000003494 hepatocyte Anatomy 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 108010040838 lymphocyte-specific protein p50 Proteins 0.000 claims 2
- 201000011278 ornithine carbamoyltransferase deficiency Diseases 0.000 claims 2
- XUIIKFGFIJCVMT-UHFFFAOYSA-N thyroxine-binding globulin Natural products IC1=CC(CC([NH3+])C([O-])=O)=CC(I)=C1OC1=CC(I)=C(O)C(I)=C1 XUIIKFGFIJCVMT-UHFFFAOYSA-N 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 230000007882 cirrhosis Effects 0.000 claims 1
- 208000019425 cirrhosis of liver Diseases 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 230000004761 fibrosis Effects 0.000 claims 1
- 206010073071 hepatocellular carcinoma Diseases 0.000 claims 1
- 231100000844 hepatocellular carcinoma Toxicity 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020054236A JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461950157P | 2014-03-09 | 2014-03-09 | |
| US61/950,157 | 2014-03-09 | ||
| PCT/US2015/019513 WO2015138348A1 (en) | 2014-03-09 | 2015-03-09 | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020054236A Division JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017512466A JP2017512466A (ja) | 2017-05-25 |
| JP2017512466A5 true JP2017512466A5 (enExample) | 2018-03-29 |
| JP6822841B2 JP6822841B2 (ja) | 2021-01-27 |
Family
ID=52737408
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016556768A Active JP6822841B2 (ja) | 2014-03-09 | 2015-03-09 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
| JP2020054236A Active JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020054236A Active JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Country Status (22)
| Country | Link |
|---|---|
| US (6) | US10167454B2 (enExample) |
| EP (2) | EP3116900B1 (enExample) |
| JP (2) | JP6822841B2 (enExample) |
| KR (1) | KR102390075B1 (enExample) |
| AU (2) | AU2015229750A1 (enExample) |
| BR (2) | BR112016020688B1 (enExample) |
| CA (1) | CA2939950C (enExample) |
| CL (2) | CL2016002235A1 (enExample) |
| CY (1) | CY1123378T1 (enExample) |
| DK (1) | DK3116900T3 (enExample) |
| ES (1) | ES2821938T3 (enExample) |
| HR (1) | HRP20201544T1 (enExample) |
| HU (1) | HUE051311T2 (enExample) |
| IL (2) | IL247329B (enExample) |
| LT (1) | LT3116900T (enExample) |
| MX (1) | MX377804B (enExample) |
| PL (1) | PL3116900T3 (enExample) |
| PT (1) | PT3116900T (enExample) |
| RS (1) | RS60902B1 (enExample) |
| SI (1) | SI3116900T1 (enExample) |
| SM (1) | SMT202000531T1 (enExample) |
| WO (2) | WO2015138357A2 (enExample) |
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| BR112015027288B1 (pt) | 2013-05-03 | 2022-03-15 | Selecta Biosciences, Inc | Composição que compreende um inibidor de mtor ligado a nanocarreadores sintéticos poliméricos |
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| HUE051311T2 (hu) * | 2014-03-09 | 2021-03-01 | Univ Pennsylvania | Ornitin transzkarbamiláz (TC) deficiencia kezelésében alkalmas készítmények |
| AU2015311707B2 (en) | 2014-09-07 | 2021-12-09 | Selecta Biosciences, Inc. | Methods and compositions for attenuating gene expression modulating anti-viral transfer vector immune responses |
| SG10202007103TA (en) | 2014-11-05 | 2020-09-29 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| US11219634B2 (en) | 2015-01-21 | 2022-01-11 | Genevant Sciences Gmbh | Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells |
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| CN108977452B (zh) | 2017-05-31 | 2022-07-19 | 北卡罗来纳大学教堂山分校 | 优化的人凝血因子ix基因表达盒及其应用 |
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| MX2020011937A (es) | 2018-05-09 | 2021-01-29 | Biomarin Pharm Inc | Metodos de tratamiento de fenilcetonuria. |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| MA52631A (fr) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics Inc | Compositions et méthodes pour le traitement de la maladie de parkinson |
| US20210220449A1 (en) * | 2018-05-15 | 2021-07-22 | Translate Bio, Inc. | Subcutaneous Delivery of Messenger RNA |
| US20200038462A1 (en) * | 2018-07-16 | 2020-02-06 | Selecta Biosciences, Inc. | Methods and compositions of otc constructs and vectors |
| AU2019304569B2 (en) | 2018-07-17 | 2023-07-06 | Helixmith Co., Ltd | Treatment of neuropathy with DNA constructs expressing IGF-1 isoforms |
| CN113227385B (zh) * | 2018-08-09 | 2024-12-24 | 比奥维拉迪维治疗股份有限公司 | 核酸分子及其用于非病毒基因疗法的用途 |
| JP7528066B2 (ja) | 2018-09-28 | 2024-08-05 | ボイジャー セラピューティクス インコーポレイテッド | 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法 |
| EP4299750A3 (en) * | 2018-12-06 | 2024-07-10 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
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| JP2023516676A (ja) * | 2020-03-03 | 2023-04-20 | アークトゥラス・セラピューティクス・インコーポレイテッド | オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法 |
| KR20220149588A (ko) * | 2020-03-04 | 2022-11-08 | 포세이다 테라퓨틱스, 인크. | 대사성 간 장애의 치료를 위한 조성물 및 방법 |
| JP2023518415A (ja) * | 2020-03-19 | 2023-05-01 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 組換えａａｖにおいてｃａｐおよびｒｅｐ配列の逆パッケージングを低減するための組成物および方法 |
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| KR20240134878A (ko) * | 2022-01-21 | 2024-09-10 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 오르니틴 트랜스카르바밀라제(otc) 결핍증을 치료하는 방법 |
| AU2023360709A1 (en) * | 2022-10-11 | 2025-05-22 | Pacific Northwest Research Institute | Constructs relating to genetic disorders |
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| HUE051311T2 (hu) | 2014-03-09 | 2021-03-01 | Univ Pennsylvania | Ornitin transzkarbamiláz (TC) deficiencia kezelésében alkalmas készítmények |
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- 2015-03-09 JP JP2016556768A patent/JP6822841B2/ja active Active
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