JP2018533976A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018533976A5 JP2018533976A5 JP2018536695A JP2018536695A JP2018533976A5 JP 2018533976 A5 JP2018533976 A5 JP 2018533976A5 JP 2018536695 A JP2018536695 A JP 2018536695A JP 2018536695 A JP2018536695 A JP 2018536695A JP 2018533976 A5 JP2018533976 A5 JP 2018533976A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- subunit
- item
- hglyra1
- composition
- 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
- 239000013598 vector Substances 0.000 claims description 105
- 238000000034 method Methods 0.000 claims description 80
- 102000039446 nucleic acids Human genes 0.000 claims description 30
- 108020004707 nucleic acids Proteins 0.000 claims description 30
- 150000007523 nucleic acids Chemical class 0.000 claims description 30
- 108010076533 Glycine Receptors Proteins 0.000 claims description 24
- 102000011714 Glycine Receptors Human genes 0.000 claims description 24
- 239000000556 agonist Substances 0.000 claims description 15
- 241000124008 Mammalia Species 0.000 claims description 14
- 210000004027 cell Anatomy 0.000 claims description 12
- 239000003814 drug Substances 0.000 claims description 12
- 229940124597 therapeutic agent Drugs 0.000 claims description 11
- 208000002193 Pain Diseases 0.000 claims description 10
- 239000013603 viral vector Substances 0.000 claims description 9
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 8
- 206010061218 Inflammation Diseases 0.000 claims description 8
- 230000004054 inflammatory process Effects 0.000 claims description 8
- 101000575685 Homo sapiens Synembryn-B Proteins 0.000 claims description 7
- 102100026014 Synembryn-B Human genes 0.000 claims description 7
- 101000821100 Homo sapiens Synapsin-1 Proteins 0.000 claims description 5
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
- 239000013607 AAV vector Substances 0.000 claims description 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 3
- 230000000694 effects Effects 0.000 claims description 3
- 238000001727 in vivo Methods 0.000 claims description 3
- 210000004962 mammalian cell Anatomy 0.000 claims description 3
- 239000005022 packaging material Substances 0.000 claims description 3
- 239000013609 scAAV vector Substances 0.000 claims description 3
- 239000013644 scAAV2 vector Substances 0.000 claims description 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 241000991587 Enterovirus C Species 0.000 claims description 2
- 241000713666 Lentivirus Species 0.000 claims description 2
- 239000013646 rAAV2 vector Substances 0.000 claims description 2
- 241000701161 unidentified adenovirus Species 0.000 claims description 2
- 101100068851 Rattus norvegicus Glra1 gene Proteins 0.000 claims 3
- 239000002431 glycine receptor agonist Substances 0.000 claims 1
- 102000004310 Ion Channels Human genes 0.000 description 19
- 108090000862 Ion Channels Proteins 0.000 description 19
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 14
- 201000010099 disease Diseases 0.000 description 13
- 230000035772 mutation Effects 0.000 description 13
- 229960003692 gamma aminobutyric acid Drugs 0.000 description 8
- BTCSSZJGUNDROE-UHFFFAOYSA-N gamma-aminobutyric acid Chemical compound NCCCC(O)=O BTCSSZJGUNDROE-UHFFFAOYSA-N 0.000 description 8
- 108050006905 Glutamate-Gated Chloride Channel Proteins 0.000 description 5
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Natural products NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 5
- 239000004471 Glycine Substances 0.000 description 4
- 206010030043 Ocular hypertension Diseases 0.000 description 4
- 229940125516 allosteric modulator Drugs 0.000 description 4
- 102000043253 matrix Gla protein Human genes 0.000 description 4
- 108010057546 matrix Gla protein Proteins 0.000 description 4
- 230000001148 spastic effect Effects 0.000 description 4
- 102000011045 Chloride Channels Human genes 0.000 description 3
- 108010062745 Chloride Channels Proteins 0.000 description 3
- 102100036912 Desmin Human genes 0.000 description 3
- 108010044052 Desmin Proteins 0.000 description 3
- 210000005045 desmin Anatomy 0.000 description 3
- 125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 description 3
- 210000003205 muscle Anatomy 0.000 description 3
- 230000000069 prophylactic effect Effects 0.000 description 3
- 230000001225 therapeutic effect Effects 0.000 description 3
- 241000701022 Cytomegalovirus Species 0.000 description 2
- 108010005551 GABA Receptors Proteins 0.000 description 2
- 102000005915 GABA Receptors Human genes 0.000 description 2
- 102000017690 GABRB1 Human genes 0.000 description 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 description 2
- 101001001362 Homo sapiens Gamma-aminobutyric acid receptor subunit beta-1 Proteins 0.000 description 2
- 241000701024 Human betaherpesvirus 5 Species 0.000 description 2
- 102000001435 Synapsin Human genes 0.000 description 2
- 108050009621 Synapsin Proteins 0.000 description 2
- 239000003795 chemical substances by application Substances 0.000 description 2
- 206010001497 Agitation Diseases 0.000 description 1
- 208000006820 Arthralgia Diseases 0.000 description 1
- 102000012286 Chitinases Human genes 0.000 description 1
- 108010022172 Chitinases Proteins 0.000 description 1
- 208000000094 Chronic Pain Diseases 0.000 description 1
- 101000906647 Drosophila melanogaster Chloride channel protein 2 Proteins 0.000 description 1
- 101500023984 Drosophila melanogaster Synapsin-1 Proteins 0.000 description 1
- 102000017696 GABRA1 Human genes 0.000 description 1
- 102000017706 GABRD Human genes 0.000 description 1
- 102000017705 GABRE Human genes 0.000 description 1
- 102000017704 GABRG1 Human genes 0.000 description 1
- 102000017703 GABRG2 Human genes 0.000 description 1
- 102000017702 GABRG3 Human genes 0.000 description 1
- 102000017700 GABRP Human genes 0.000 description 1
- 102000016407 GABRQ Human genes 0.000 description 1
- 102000016406 GABRR1 Human genes 0.000 description 1
- 102000016405 GABRR2 Human genes 0.000 description 1
- 108060004404 GABRR2 Proteins 0.000 description 1
- 102000016404 GABRR3 Human genes 0.000 description 1
- 108700039691 Genetic Promoter Regions Proteins 0.000 description 1
- 208000010412 Glaucoma Diseases 0.000 description 1
- 101000893331 Homo sapiens Gamma-aminobutyric acid receptor subunit alpha-1 Proteins 0.000 description 1
- 101001073587 Homo sapiens Gamma-aminobutyric acid receptor subunit delta Proteins 0.000 description 1
- 101001073581 Homo sapiens Gamma-aminobutyric acid receptor subunit epsilon Proteins 0.000 description 1
- 101001073577 Homo sapiens Gamma-aminobutyric acid receptor subunit gamma-1 Proteins 0.000 description 1
- 101000926813 Homo sapiens Gamma-aminobutyric acid receptor subunit gamma-2 Proteins 0.000 description 1
- 101000926819 Homo sapiens Gamma-aminobutyric acid receptor subunit gamma-3 Proteins 0.000 description 1
- 101000822394 Homo sapiens Gamma-aminobutyric acid receptor subunit pi Proteins 0.000 description 1
- 101000822386 Homo sapiens Gamma-aminobutyric acid receptor subunit rho-1 Proteins 0.000 description 1
- 101000822408 Homo sapiens Gamma-aminobutyric acid receptor subunit rho-3 Proteins 0.000 description 1
- 101000822412 Homo sapiens Gamma-aminobutyric acid receptor subunit theta Proteins 0.000 description 1
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 description 1
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 description 1
- 101100068013 Rattus norvegicus Gabrb1 gene Proteins 0.000 description 1
- 101000757149 Rhizopus oryzae Glucoamylase 1 Proteins 0.000 description 1
- 230000003281 allosteric effect Effects 0.000 description 1
- 206010003246 arthritis Diseases 0.000 description 1
- 208000035475 disorder Diseases 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 238000002651 drug therapy Methods 0.000 description 1
- 239000012634 fragment Substances 0.000 description 1
- 208000018937 joint inflammation Diseases 0.000 description 1
- 208000004296 neuralgia Diseases 0.000 description 1
- 208000021722 neuropathic pain Diseases 0.000 description 1
- 108090000623 proteins and genes Proteins 0.000 description 1
- 102000005962 receptors Human genes 0.000 description 1
- 108020003175 receptors Proteins 0.000 description 1
- 230000035945 sensitivity Effects 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562235914P | 2015-10-01 | 2015-10-01 | |
| US201562235920P | 2015-10-01 | 2015-10-01 | |
| US62/235,920 | 2015-10-01 | ||
| US62/235,914 | 2015-10-01 | ||
| US201662303907P | 2016-03-04 | 2016-03-04 | |
| US62/303,907 | 2016-03-04 | ||
| US201662378509P | 2016-08-23 | 2016-08-23 | |
| US62/378,509 | 2016-08-23 | ||
| PCT/US2016/054199 WO2017058926A1 (en) | 2015-10-01 | 2016-09-28 | Targeted expression of chloride channels and methods of use thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018533976A JP2018533976A (ja) | 2018-11-22 |
| JP2018533976A5 true JP2018533976A5 (enExample) | 2019-10-31 |
| JP7097070B2 JP7097070B2 (ja) | 2022-07-07 |
Family
ID=58427328
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018536695A Active JP7097070B2 (ja) | 2015-10-01 | 2016-09-28 | クロライドチャネルの標的発現及びその使用方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US11407812B2 (enExample) |
| EP (1) | EP3355938B1 (enExample) |
| JP (1) | JP7097070B2 (enExample) |
| KR (2) | KR20250012721A (enExample) |
| CN (1) | CN108289965A (enExample) |
| AU (1) | AU2016332847B2 (enExample) |
| BR (1) | BR112018006480A2 (enExample) |
| CA (1) | CA2999279A1 (enExample) |
| HK (1) | HK1252915A1 (enExample) |
| SG (1) | SG10201912703PA (enExample) |
| WO (1) | WO2017058926A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12496274B2 (en) | 2018-11-14 | 2025-12-16 | Flagship Pioneering Innovations V, Inc. | Compositions and methods for compartment-specific cargo delivery |
| CN113613633A (zh) * | 2018-11-14 | 2021-11-05 | 旗舰先锋创新V股份有限公司 | 用于cns递送的融合剂脂质体组合物 |
| CN110679548B (zh) * | 2019-10-24 | 2021-11-30 | 南方医科大学南方医院 | 一种自闭症小鼠模型的构建方法 |
| CN111759857A (zh) * | 2020-06-30 | 2020-10-13 | 宁波市微循环与莨菪类药研究所 | Gabrd甲基化靶点在制备抗海洛因复吸药物中的应用 |
| CN115480485B (zh) * | 2022-09-14 | 2024-09-10 | 南开大学 | 一种基于数据驱动模型的软镜扭转运动安全控制方法 |
| GB202401492D0 (en) | 2024-02-05 | 2024-03-20 | Ucl Business Ltd | Chloride channels and uses thereof |
| CN119331917B (zh) * | 2024-10-28 | 2025-10-17 | 北京爱思益普生物科技股份有限公司 | 能够表达GlyR α1/β型GlyR的细胞株及其制备方法 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3644643A (en) * | 1969-10-13 | 1972-02-22 | Unimed Inc | Method of reducing intraocular pressure using glycine |
| US4559157A (en) | 1983-04-21 | 1985-12-17 | Creative Products Resource Associates, Ltd. | Cosmetic applicator useful for skin moisturizing |
| LU84979A1 (fr) | 1983-08-30 | 1985-04-24 | Oreal | Composition cosmetique ou pharmaceutique sous forme aqueuse ou anhydre dont la phase grasse contient un polyether oligomere et polyethers oligomeres nouveaux |
| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| US4820508A (en) | 1987-06-23 | 1989-04-11 | Neutrogena Corporation | Skin protective composition |
| US4992478A (en) | 1988-04-04 | 1991-02-12 | Warner-Lambert Company | Antiinflammatory skin moisturizing composition and method of preparing same |
| US5527703A (en) * | 1994-05-25 | 1996-06-18 | Merck & Co., Inc. | DNA encoding glutamate gated chloride channels |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6464986B1 (en) * | 2000-04-14 | 2002-10-15 | Allegan Sales, Inc. | Method for treating pain by peripheral administration of a neurotoxin |
| DE60139398D1 (de) * | 2000-06-20 | 2009-09-10 | Bionomics Ltd | Mutation, welche mit epilepsie assoziiert ist |
| AU2002250076A1 (en) * | 2001-02-16 | 2002-09-04 | Bristol-Myers Squibb Company | Polynucleotides encoding a novel glycine receptor alpha subunit expressed in the gastrointestinal tract, hgra4, and splice variant thereof |
| US20040058313A1 (en) | 2002-04-24 | 2004-03-25 | Abreu Marcio Marc | Compositions, targets, methods and devices for the therapy of ocular and periocular disorders |
| US8298818B2 (en) * | 2006-04-28 | 2012-10-30 | University Of Florida Research Foundation, Inc. | Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter |
| CN101522903B (zh) | 2006-08-24 | 2013-07-31 | 威洛克有限公司 | 具有重叠开放阅读框的基因在昆虫细胞中的表达及其方法和组合物 |
| US20080289058A1 (en) * | 2007-05-14 | 2008-11-20 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Targeted delivery of glycine receptors to excitable cells |
| EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| WO2009133191A1 (en) * | 2008-04-30 | 2009-11-05 | Dublin City University | Compositions and methods for expressing in-frame multimeric proteins |
| WO2010042799A2 (en) | 2008-10-09 | 2010-04-15 | Howard Hughes Medical Institute | Novel chimeric ligand-gated ion channels and methods of use thereof |
| WO2011137210A2 (en) | 2010-04-30 | 2011-11-03 | The Regents Of The University Of California | Modulating compliance of trabecular meshwork |
| TW201306825A (zh) | 2011-05-11 | 2013-02-16 | Kirax Corp | 狀況治療之改良套裝 |
| CN105188767A (zh) | 2012-07-25 | 2015-12-23 | 布罗德研究所有限公司 | 可诱导的dna结合蛋白和基因组干扰工具及其应用 |
| EP2692868A1 (en) | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
| US9624288B2 (en) * | 2012-12-10 | 2017-04-18 | University of Pittsburgh—of the Commonwealth System of Higher Education | Engineered receptors and their use |
| JP2017501834A (ja) | 2014-01-08 | 2017-01-19 | サーキット セラピューティクス， インコーポレイテッド | 高眼圧症の治療管理のための方法 |
| US20150283398A1 (en) | 2014-03-27 | 2015-10-08 | Circuit Therapeutics, Inc. | System and method for therapeutic management of cough |
| AU2015330092A1 (en) | 2014-10-06 | 2017-04-13 | Arthrogen B.V. | AAV-based gene therapy |
| AU2016324317A1 (en) * | 2015-09-17 | 2018-03-08 | Coda Biotherapeutics, Inc. | Compositions and methods for treating neurological disorders |
| GB2547179A (en) | 2015-10-26 | 2017-08-16 | Quethera Ltd | Genetic construct |
| AU2017254652B2 (en) | 2016-04-21 | 2022-12-01 | Virovek, Inc. | AAV production in insect cells, methods and compositions therefor |
-
2016
- 2016-09-28 HK HK18112264.4A patent/HK1252915A1/zh unknown
- 2016-09-28 BR BR112018006480A patent/BR112018006480A2/pt not_active IP Right Cessation
- 2016-09-28 SG SG10201912703PA patent/SG10201912703PA/en unknown
- 2016-09-28 CN CN201680068232.8A patent/CN108289965A/zh active Pending
- 2016-09-28 JP JP2018536695A patent/JP7097070B2/ja active Active
- 2016-09-28 US US15/764,733 patent/US11407812B2/en active Active
- 2016-09-28 WO PCT/US2016/054199 patent/WO2017058926A1/en not_active Ceased
- 2016-09-28 KR KR1020257000627A patent/KR20250012721A/ko active Pending
- 2016-09-28 AU AU2016332847A patent/AU2016332847B2/en active Active
- 2016-09-28 EP EP16852497.3A patent/EP3355938B1/en active Active
- 2016-09-28 CA CA2999279A patent/CA2999279A1/en not_active Abandoned
- 2016-09-28 KR KR1020187012245A patent/KR20180072713A/ko not_active Ceased
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018533976A5 (enExample) | ||
| US20220331409A1 (en) | Factor ix gene therapy | |
| JP7607007B2 (ja) | 変異ａａｖ、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法 | |
| KR102527259B1 (ko) | 스터퍼/필러 폴리누클레오티드 서열을 포함하는 벡터 및 사용 방법 | |
| JP6805174B2 (ja) | 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質 | |
| KR102057540B1 (ko) | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 | |
| CN107002096A (zh) | 基于aav的基因疗法 | |
| US12246072B2 (en) | Gene therapy for the treatment of CNGB1-linked retinitis pigmentosa | |
| JP7558535B2 (ja) | アルデヒドデヒドロゲナーゼ欠損症の治療のための遺伝子治療 | |
| CN104936606A (zh) | 糖原贮积病的基因疗法 | |
| CN120324641A (zh) | 用于治疗达农病的基因治疗载体 | |
| EP3740244B1 (en) | Adeno-associated virus gene therapy for 21-hydroxylase deficiency | |
| JP2021520811A (ja) | ヘキソサミニダーゼアルファおよびベータサブユニットをコードしているバイシストロニックａａｖベクターならびにその使用 | |
| AU2021358413A1 (en) | Nucleic acid constructs, viral vectors and viral particles | |
| RU2837753C2 (ru) | Вариант aav, композиции и способы, в которых он используется, а также способы его применения для переноса генов в клетки, органы и ткани | |
| WO2023156530A1 (en) | Gene therapy for neurodegenerative diseases | |
| Gregorevic et al. | Functional enhancement of skeletal muscle by gene transfer | |
| KR20240052746A (ko) | Kcnv2 유전자 요법 | |
| BR112016001210B1 (pt) | Partículas de aav recombinates, e composição farmacêutica | |
| BR112017027956B1 (pt) | Vetores de vírus adenoassociado recombinante, uso e método de produção dos mesmos, e composição farmacêutica |