JP2018522595A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
Links
- 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 (cg-RX-API-DMAC10.html) | 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 (cg-RX-API-DMAC10.html) |
| EP (1) | EP3331570A1 (cg-RX-API-DMAC10.html) |
| JP (1) | JP2018522595A (cg-RX-API-DMAC10.html) |
| KR (1) | KR20180034467A (cg-RX-API-DMAC10.html) |
| CN (1) | CN108136048A (cg-RX-API-DMAC10.html) |
| CA (1) | CA2992511A1 (cg-RX-API-DMAC10.html) |
| RU (1) | RU2018104098A (cg-RX-API-DMAC10.html) |
| WO (1) | WO2017021359A1 (cg-RX-API-DMAC10.html) |
Families Citing this family (19)
| 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 |
| KR20210151785A (ko) * | 2019-03-13 | 2021-12-14 | 제너레이션 바이오 컴퍼니 | 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 |
| 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 |
| EP4107259A1 (en) * | 2020-02-18 | 2022-12-28 | Vrije Universiteit Brussel | Novel combination of nucleic acid regulatory elements and methods and uses thereof |
| CN115515646A (zh) | 2020-03-04 | 2022-12-23 | 波赛达治疗公司 | 用于治疗代谢性肝病的组合物和方法 |
| WO2021222653A1 (en) * | 2020-04-29 | 2021-11-04 | Saliogen Therapeutics, Inc. | Compositions and methods for treatment of familial hypercholesterolemia and elevated low-density lipoprotein cholesterol |
| CA3187002A1 (en) * | 2020-07-15 | 2022-01-20 | Danmarks Tekniske Universitet | Therapeutic microbes |
| GB202107473D0 (en) * | 2021-05-26 | 2021-07-07 | Univ Denmark Tech Dtu | Therapeutic microbes |
| IL301255A (en) * | 2020-10-09 | 2023-05-01 | UCB Biopharma SRL | Nucleic acid structures, viral vectors and viral particles |
| GB2601752A (en) * | 2020-12-08 | 2022-06-15 | Maavrx Ltd | Expression vector |
| WO2023012313A1 (en) * | 2021-08-04 | 2023-02-09 | Genethon | Hybrid promoters for gene expression in muscles and in the cns |
| WO2023049874A1 (en) * | 2021-09-24 | 2023-03-30 | Duke University | Compositions for and methods of 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 |
| DE3681787D1 (de) | 1985-07-05 | 1991-11-07 | Whitehead Biomedical Inst | Expression von fremdem genetischem material in epithelzellen. |
| HK1008049A1 (en) | 1987-09-11 | 1999-04-30 | Whitehead Institute For Biomedical Research | Transduced fibroblasts and use therefor |
| 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 |
| CA2329259C (en) | 1998-05-27 | 2003-08-05 | Avigen, Inc. | Convection-enhanced delivery of aav vectors |
| GB0024550D0 (cg-RX-API-DMAC10.html) * | 2000-10-06 | 2000-11-22 | Oxford Biomedica Ltd | |
| KR100456062B1 (ko) * | 2001-06-18 | 2004-11-08 | 박영식 | 재조합 대장균에 의한 테리딘 화합물의 제조방법 |
| AU2010316982B2 (en) * | 2009-11-09 | 2013-09-12 | Genepod Therapeutics Ab | Novel viral vector construct for neuron specific continuous DOPA synthesis in vivo |
| GB201118636D0 (en) * | 2011-10-28 | 2011-12-07 | Oxford Biomedica Ltd | Nucleotide sequence |
-
2016
- 2016-08-01 WO PCT/EP2016/068315 patent/WO2017021359A1/en not_active Ceased
- 2016-08-01 RU RU2018104098A patent/RU2018104098A/ru not_active Application Discontinuation
- 2016-08-01 US US15/748,145 patent/US20190032079A1/en not_active Abandoned
- 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 CN CN201680045737.2A patent/CN108136048A/zh active Pending
- 2016-08-01 KR KR1020187003356A patent/KR20180034467A/ko not_active Withdrawn
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20220127621A1 (en) | Fusion proteins and fusion ribonucleic acids for tracking and manipulating cellular rna | |
| CA3042358C (en) | Zinc fingers and related adenovirus-associated virus vectors for the treatment of polyglutamine diseases, such as Huntington's disease | |
| JP2024012479A (ja) | 転写調節要素及びその使用 | |
| CN116096734A (zh) | Aav衣壳的向性的重定向 | |
| CA2985372C (en) | Polynucleotides, vectors and methods for insertion and expression of transgenes | |
| CA3061368A1 (en) | Compositions and methods of treating huntington's disease | |
| CN114450031A (zh) | 靶向rna的敲低和替代组合物及使用方法 | |
| JP2013509168A5 (cg-RX-API-DMAC10.html) | ||
| JP2022507402A (ja) | 肝特異的ウイルスプロモーター及びその使用方法 | |
| HRP20241180T1 (hr) | Modificirani faktor ix, te pripravci, postupci i upotreba za prijenos gena u stanice, organe i tkiva | |
| FI3546585T3 (fi) | Muokatun intronin käsittäviä nukleiinihappoja käytettäviksi hyperbilirubinemian hoidossa | |
| JP2011062204A5 (cg-RX-API-DMAC10.html) | ||
| RU2018104098A (ru) | Системный синтез и регуляция l-дофа | |
| JP2020054367A (ja) | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 | |
| KR20210096168A (ko) | 신경퇴행성 질환을 위한 유전자 요법 | |
| RU2012123943A (ru) | Новая вирусная векторная конструкция для нейронспецифического оптимизированного непрерывного синтеза dopa in vivo | |
| CA3165624A1 (en) | Gene therapy for neurodegenerative disorders using polynucleotide silencing and replacement | |
| HRP20240184T1 (hr) | Polinukleotidi | |
| TW202421787A (zh) | 保護心臟之心臟病療法 | |
| JP2020535804A5 (cg-RX-API-DMAC10.html) | ||
| AU2022267320A9 (en) | Multiplex crispr/cas9-mediated target gene activation system | |
| JP2023535808A5 (cg-RX-API-DMAC10.html) | ||
| JP2023504448A (ja) | 神経変性障害に対する遺伝子療法 | |
| WO2020210724A1 (en) | Htra1 modulation for treatment of amd | |
| JP2021529519A5 (cg-RX-API-DMAC10.html) |