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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- nucleic acid
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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 (cg-RX-API-DMAC7.html) | 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)
Families Citing this family (41)
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| RU2020109343A (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| CN114642735A (zh) | 2015-01-21 | 2022-06-21 | 菲泽尔克斯公司 | 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统 |
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| AU2020216480A1 (en) | 2019-01-31 | 2021-09-09 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of AAV capsids |
| 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 |
| CA3169529A1 (en) * | 2020-03-04 | 2021-09-10 | Jingjing Jiang | Compositions and methods for the treatment of metabolic liver disorders |
| EP4121544A1 (en) * | 2020-03-19 | 2023-01-25 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| BR112022022212A2 (pt) | 2020-05-12 | 2022-12-13 | Univ Pennsylvania | Composições para redução específica de drg de expressão de transgene |
| JP2024505257A (ja) | 2021-02-01 | 2024-02-05 | レジェンクスバイオ インコーポレーテッド | 神経セロイドリポフスチン症の遺伝子治療 |
| WO2022224372A1 (ja) * | 2021-04-21 | 2022-10-27 | 学校法人自治医科大学 | オルニチントランスカルバミラーゼ欠損症の治療用アデノ随伴ウイルスビリオン |
| WO2023028455A1 (en) * | 2021-08-23 | 2023-03-02 | Bioverativ Therapeutics Inc. | Closed-end dna production with inverted terminal repeat sequences |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20250161493A1 (en) * | 2022-01-21 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions and methods for in vivo nuclease-mediated treatment of ornithine transcarbamylase (otc) deficiency |
| CA3268024A1 (en) * | 2022-10-11 | 2024-04-18 | Pacific Northwest Research Institute | CONSTRUCTIONS RELATED TO GENETIC DISORDERS |
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