JP2018522595A5 - - Google Patents
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- JP2018522595A5 JP2018522595A5 JP2018526302A JP2018526302A JP2018522595A5 JP 2018522595 A5 JP2018522595 A5 JP 2018522595A5 JP 2018526302 A JP2018526302 A JP 2018526302A JP 2018526302 A JP2018526302 A JP 2018526302A JP 2018522595 A5 JP2018522595 A5 JP 2018522595A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- polypeptide
- group
- expression system
- polynucleotide
- 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.)
- Pending
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- 108090000765 processed proteins & peptides Proteins 0.000 claims 36
- 229920001184 polypeptide Polymers 0.000 claims 34
- 102000004196 processed proteins & peptides Human genes 0.000 claims 34
- 108091000117 Tyrosine 3-Monooxygenase Proteins 0.000 claims 19
- 102000048218 Tyrosine 3-monooxygenases Human genes 0.000 claims 19
- 108091033319 polynucleotide Proteins 0.000 claims 17
- 239000002157 polynucleotide Substances 0.000 claims 17
- 102000040430 polynucleotide Human genes 0.000 claims 17
- 102100027346 GTP cyclohydrolase 1 Human genes 0.000 claims 12
- 239000012634 fragment Substances 0.000 claims 12
- 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 11
- 101710094136 GTP cyclohydrolase 1 Proteins 0.000 claims 11
- 102100028505 6-pyruvoyl tetrahydrobiopterin synthase Human genes 0.000 claims 10
- 108010045523 6-pyruvoyltetrahydropterin synthase Proteins 0.000 claims 10
- 208000018737 Parkinson disease Diseases 0.000 claims 6
- 239000013598 vector Substances 0.000 claims 6
- 210000004027 cell Anatomy 0.000 claims 5
- VYFYYTLLBUKUHU-UHFFFAOYSA-N dopamine Chemical compound NCCC1=CC=C(O)C(O)=C1 VYFYYTLLBUKUHU-UHFFFAOYSA-N 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 230000001105 regulatory effect Effects 0.000 claims 4
- 108020004684 Internal Ribosome Entry Sites Proteins 0.000 claims 3
- 230000004071 biological effect Effects 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 230000002255 enzymatic effect Effects 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 230000001124 posttranscriptional effect Effects 0.000 claims 3
- 208000012661 Dyskinesia Diseases 0.000 claims 2
- WTDRDQBEARUVNC-LURJTMIESA-N L-DOPA Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C(O)=C1 WTDRDQBEARUVNC-LURJTMIESA-N 0.000 claims 2
- WTDRDQBEARUVNC-UHFFFAOYSA-N L-Dopa Natural products OC(=O)C(N)CC1=CC=C(O)C(O)=C1 WTDRDQBEARUVNC-UHFFFAOYSA-N 0.000 claims 2
- ZPUCINDJVBIVPJ-LJISPDSOSA-N cocaine Chemical compound O([C@H]1C[C@@H]2CC[C@@H](N2C)[C@H]1C(=O)OC)C(=O)C1=CC=CC=C1 ZPUCINDJVBIVPJ-LJISPDSOSA-N 0.000 claims 2
- 230000007812 deficiency Effects 0.000 claims 2
- 229960003638 dopamine Drugs 0.000 claims 2
- WBJZXBUVECZHCE-UHFFFAOYSA-N dyspropterin Chemical compound N1=C(N)NC(=O)C2=C1NCC(C(=O)C(=O)C)N2 WBJZXBUVECZHCE-UHFFFAOYSA-N 0.000 claims 2
- 239000013612 plasmid Substances 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 206010001367 Adrenal insufficiency Diseases 0.000 claims 1
- 208000006096 Attention Deficit Disorder with Hyperactivity Diseases 0.000 claims 1
- 208000036864 Attention deficit/hyperactivity disease Diseases 0.000 claims 1
- 208000015798 BH4-deficient hyperphenylalaninemia B Diseases 0.000 claims 1
- 108010078791 Carrier Proteins Proteins 0.000 claims 1
- 102000014914 Carrier Proteins Human genes 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 206010012289 Dementia Diseases 0.000 claims 1
- 208000002603 Dopa-responsive dystonia Diseases 0.000 claims 1
- 208000014094 Dystonic disease Diseases 0.000 claims 1
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 claims 1
- 208000001613 Gambling Diseases 0.000 claims 1
- 101000862581 Homo sapiens GTP cyclohydrolase 1 Proteins 0.000 claims 1
- 206010020772 Hypertension Diseases 0.000 claims 1
- 208000001953 Hypotension Diseases 0.000 claims 1
- 208000009829 Lewy Body Disease Diseases 0.000 claims 1
- 201000002832 Lewy body dementia Diseases 0.000 claims 1
- 208000005565 Marijuana Use Diseases 0.000 claims 1
- 208000001089 Multiple system atrophy Diseases 0.000 claims 1
- 101100228519 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) gch-1 gene Proteins 0.000 claims 1
- 206010034010 Parkinsonism Diseases 0.000 claims 1
- 206010034158 Pathological gambling Diseases 0.000 claims 1
- 102100034333 Synaptic vesicular amine transporter Human genes 0.000 claims 1
- 101710164184 Synaptic vesicular amine transporter Proteins 0.000 claims 1
- 206010068100 Vascular parkinsonism Diseases 0.000 claims 1
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims 1
- 230000005856 abnormality Effects 0.000 claims 1
- 239000002671 adjuvant Substances 0.000 claims 1
- 208000017515 adrenocortical insufficiency Diseases 0.000 claims 1
- 125000000539 amino acid group Chemical group 0.000 claims 1
- 230000003197 catalytic effect Effects 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 230000001684 chronic effect Effects 0.000 claims 1
- 230000037326 chronic stress Effects 0.000 claims 1
- 229960003920 cocaine Drugs 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 108700006189 dopamine beta hydroxylase deficiency Proteins 0.000 claims 1
- 208000009308 dopamine beta-hydroxylase deficiency Diseases 0.000 claims 1
- 208000010118 dystonia Diseases 0.000 claims 1
- 201000006517 essential tremor Diseases 0.000 claims 1
- 230000002068 genetic effect Effects 0.000 claims 1
- 230000036543 hypotension Effects 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 229940005483 opioid analgesics Drugs 0.000 claims 1
- 208000028173 post-traumatic stress disease Diseases 0.000 claims 1
- 201000002212 progressive supranuclear palsy Diseases 0.000 claims 1
- 230000001177 retroviral effect Effects 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 201000000980 schizophrenia Diseases 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 230000009885 systemic effect Effects 0.000 claims 1
- 230000002792 vascular Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562200151P | 2015-08-03 | 2015-08-03 | |
| US62/200,151 | 2015-08-03 | ||
| PCT/EP2016/068315 WO2017021359A1 (en) | 2015-08-03 | 2016-08-01 | Systemic synthesis and regulation of l-dopa |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018522595A JP2018522595A (ja) | 2018-08-16 |
| JP2018522595A5 true JP2018522595A5 (https=) | 2019-09-12 |
Family
ID=56682101
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018526302A Pending JP2018522595A (ja) | 2015-08-03 | 2016-08-01 | Ｌ−ｄｏｐａの全身合成及び調節 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20190032079A1 (https=) |
| EP (1) | EP3331570A1 (https=) |
| JP (1) | JP2018522595A (https=) |
| KR (1) | KR20180034467A (https=) |
| CN (1) | CN108136048A (https=) |
| CA (1) | CA2992511A1 (https=) |
| RU (1) | RU2018104098A (https=) |
| WO (1) | WO2017021359A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| US20210093735A1 (en) * | 2018-04-26 | 2021-04-01 | The University Of North Carolina At Chapel Hill | Methods and compositions for treatment of hemophilia |
| US12313635B2 (en) * | 2018-07-27 | 2025-05-27 | The Regents Of The University Of California | Biomarker for thoracic aortic aneurysm |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| SG11202109850SA (en) | 2019-03-13 | 2021-10-28 | Generation Bio Co | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
| KR20210148273A (ko) * | 2019-04-08 | 2021-12-07 | 제네똥 | 근육 발현을 위한 하이브리드 프로모터 |
| GB2587319A (en) * | 2019-08-12 | 2021-03-31 | Healing Genes Llc | Genetic construct |
| EP4081643A1 (en) * | 2019-12-24 | 2022-11-02 | Asklepios Biopharmaceutical, Inc. | Regulatory nucleic acid sequences |
| JP7744027B2 (ja) * | 2020-02-18 | 2025-09-25 | フレイエ ユニヴェルシテイト ブリュッセル | 核酸調節エレメントの新規な組合せ並びにその方法及び使用 |
| AU2021230361A1 (en) * | 2020-03-04 | 2022-09-08 | Poseida Therapeutics, Inc. | Compositions and methods for the treatment of Metabolic Liver Disorders |
| KR20230002594A (ko) * | 2020-04-29 | 2023-01-05 | 살리오젠 테라퓨틱스 인코포레이티드 | 가족성 고콜레스테롤혈증 및 상승된 저밀도 지단백질 콜레스테롤의 치료를 위한 조성물 및 방법 |
| EP4181937A1 (en) * | 2020-07-15 | 2023-05-24 | Danmarks Tekniske Universitet | Therapeutic microbes |
| GB2601752A (en) * | 2020-12-08 | 2022-06-15 | Maavrx Ltd | Expression vector |
| EP4381077A1 (en) * | 2021-08-04 | 2024-06-12 | Genethon | Hybrid promoters for gene expression in muscles and in the cns |
| EP4405492A4 (en) * | 2021-09-24 | 2025-07-09 | Univ Duke | COMPOSITIONS AND METHODS FOR TREATING AND/OR PREVENTING GLUTARIC ACIDURIA TYPE I |
| CN117106825B (zh) * | 2023-08-28 | 2024-11-01 | 康霖生物科技（杭州）有限公司 | 用于治疗帕金森病的基因治疗载体及其用途 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| IL79289A (en) | 1985-07-05 | 1992-01-15 | Whitehead Biomedical Inst | Introduction and expression of foreign genetic material into keratinocytes using a recombinant retrovirus |
| ATE117375T1 (de) | 1987-09-11 | 1995-02-15 | Whitehead Biomedical Inst | Transduktionsveränderte fibroblasten und ihre anwendung. |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| US5677158A (en) | 1995-06-07 | 1997-10-14 | Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6451306B1 (en) | 1998-04-15 | 2002-09-17 | The Regents Of The University Of California | Methods for therapy of neurodegenerative disease of the brain |
| WO1999061066A2 (en) | 1998-05-27 | 1999-12-02 | Avigen, Inc. | Convection-enhanced delivery of aav vectors |
| GB0024550D0 (https=) * | 2000-10-06 | 2000-11-22 | Oxford Biomedica Ltd | |
| KR100456062B1 (ko) * | 2001-06-18 | 2004-11-08 | 박영식 | 재조합 대장균에 의한 테리딘 화합물의 제조방법 |
| KR20120089743A (ko) * | 2009-11-09 | 2012-08-13 | 제네포드 테라퓨틱스 에이비 | 생체 내에서 최적화된 뉴런 특이적 연속 도파 합성을 위한 신규한 바이러스 벡터 구성물 |
| GB201118636D0 (en) * | 2011-10-28 | 2011-12-07 | Oxford Biomedica Ltd | Nucleotide sequence |
-
2016
- 2016-08-01 US US15/748,145 patent/US20190032079A1/en not_active Abandoned
- 2016-08-01 CN CN201680045737.2A patent/CN108136048A/zh active Pending
- 2016-08-01 WO PCT/EP2016/068315 patent/WO2017021359A1/en not_active Ceased
- 2016-08-01 KR KR1020187003356A patent/KR20180034467A/ko not_active Withdrawn
- 2016-08-01 JP JP2018526302A patent/JP2018522595A/ja active Pending
- 2016-08-01 CA CA2992511A patent/CA2992511A1/en not_active Abandoned
- 2016-08-01 EP EP16750423.2A patent/EP3331570A1/en not_active Withdrawn
- 2016-08-01 RU RU2018104098A patent/RU2018104098A/ru not_active Application Discontinuation
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