JP2020515292A5 - - Google Patents
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- JP2020515292A5 JP2020515292A5 JP2020502552A JP2020502552A JP2020515292A5 JP 2020515292 A5 JP2020515292 A5 JP 2020515292A5 JP 2020502552 A JP2020502552 A JP 2020502552A JP 2020502552 A JP2020502552 A JP 2020502552A JP 2020515292 A5 JP2020515292 A5 JP 2020515292A5
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- Prior art keywords
- nucleic acid
- acid cassette
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- Prior art date
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- 108020004707 nucleic acids Proteins 0.000 claims description 72
- 102000039446 nucleic acids Human genes 0.000 claims description 72
- 150000007523 nucleic acids Chemical class 0.000 claims description 72
- 108700019146 Transgenes Proteins 0.000 claims description 56
- 102000001675 Parvalbumin Human genes 0.000 claims description 48
- 108060005874 Parvalbumin Proteins 0.000 claims description 48
- 210000002569 neuron Anatomy 0.000 claims description 37
- 108090000623 proteins and genes Proteins 0.000 claims description 26
- 239000012634 fragment Substances 0.000 claims description 21
- 102000052510 DNA-Binding Proteins Human genes 0.000 claims description 15
- 101710096438 DNA-binding protein Proteins 0.000 claims description 14
- 101000631760 Homo sapiens Sodium channel protein type 1 subunit alpha Proteins 0.000 claims description 14
- 101000684813 Homo sapiens Sodium channel subunit beta-1 Proteins 0.000 claims description 14
- 101000684822 Homo sapiens Sodium channel subunit beta-2 Proteins 0.000 claims description 14
- 102100028910 Sodium channel protein type 1 subunit alpha Human genes 0.000 claims description 14
- 102100023732 Sodium channel subunit beta-1 Human genes 0.000 claims description 14
- 102100023722 Sodium channel subunit beta-2 Human genes 0.000 claims description 14
- 210000004027 cell Anatomy 0.000 claims description 14
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 14
- 230000000926 neurological effect Effects 0.000 claims description 14
- 102000004169 proteins and genes Human genes 0.000 claims description 14
- 101000648077 Homo sapiens Syntaxin-binding protein 1 Proteins 0.000 claims description 11
- 102100025293 Syntaxin-binding protein 1 Human genes 0.000 claims description 11
- 230000008045 co-localization Effects 0.000 claims description 10
- 238000010362 genome editing Methods 0.000 claims description 10
- 238000002714 localization assay Methods 0.000 claims description 10
- 239000013598 vector Substances 0.000 claims description 10
- 239000013603 viral vector Substances 0.000 claims description 6
- 208000024827 Alzheimer disease Diseases 0.000 claims description 5
- 230000004568 DNA-binding Effects 0.000 claims description 5
- 108090000862 Ion Channels Proteins 0.000 claims description 5
- 102000004310 Ion Channels Human genes 0.000 claims description 5
- 201000007547 Dravet syndrome Diseases 0.000 claims description 4
- 208000036572 Myoclonic epilepsy Diseases 0.000 claims description 4
- 206010073677 Severe myoclonic epilepsy of infancy Diseases 0.000 claims description 4
- 108010052164 Sodium Channels Proteins 0.000 claims description 4
- 102000018674 Sodium Channels Human genes 0.000 claims description 4
- 238000003556 assay Methods 0.000 claims description 4
- 230000002055 immunohistochemical effect Effects 0.000 claims description 4
- 239000002858 neurotransmitter agent Substances 0.000 claims description 4
- 230000001105 regulatory effect Effects 0.000 claims description 4
- 102000004257 Potassium Channel Human genes 0.000 claims description 3
- 208000034799 Tauopathies Diseases 0.000 claims description 3
- 230000003247 decreasing effect Effects 0.000 claims description 3
- 206010015037 epilepsy Diseases 0.000 claims description 3
- 230000004770 neurodegeneration Effects 0.000 claims description 3
- 230000008587 neuronal excitability Effects 0.000 claims description 3
- 108020001213 potassium channel Proteins 0.000 claims description 3
- 239000013607 AAV vector Substances 0.000 claims description 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 102100035902 Glutamate decarboxylase 1 Human genes 0.000 claims description 2
- 102100035857 Glutamate decarboxylase 2 Human genes 0.000 claims description 2
- 102100030087 Homeobox protein DLX-1 Human genes 0.000 claims description 2
- 102100022373 Homeobox protein DLX-5 Human genes 0.000 claims description 2
- 101000873546 Homo sapiens Glutamate decarboxylase 1 Proteins 0.000 claims description 2
- 101000873786 Homo sapiens Glutamate decarboxylase 2 Proteins 0.000 claims description 2
- 101000864690 Homo sapiens Homeobox protein DLX-1 Proteins 0.000 claims description 2
- 101000901627 Homo sapiens Homeobox protein DLX-5 Proteins 0.000 claims description 2
- 101000821100 Homo sapiens Synapsin-1 Proteins 0.000 claims description 2
- 101150111110 NKX2-1 gene Proteins 0.000 claims description 2
- 102100021905 Synapsin-1 Human genes 0.000 claims description 2
- 108700009124 Transcription Initiation Site Proteins 0.000 claims description 2
- 210000001130 astrocyte Anatomy 0.000 claims description 2
- 210000005064 dopaminergic neuron Anatomy 0.000 claims description 2
- 230000002964 excitative effect Effects 0.000 claims description 2
- 210000000274 microglia Anatomy 0.000 claims description 2
- 210000002161 motor neuron Anatomy 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 53
- 101710163270 Nuclease Proteins 0.000 description 3
- 208000012902 Nervous system disease Diseases 0.000 description 2
- 230000002103 transcriptional effect Effects 0.000 description 2
- 108091033409 CRISPR Proteins 0.000 description 1
- 108700020911 DNA-Binding Proteins Proteins 0.000 description 1
- 102000018120 Recombinases Human genes 0.000 description 1
- 108010091086 Recombinases Proteins 0.000 description 1
- 101710185494 Zinc finger protein Proteins 0.000 description 1
- 102100023597 Zinc finger protein 816 Human genes 0.000 description 1
- 239000012636 effector Substances 0.000 description 1
- 230000000415 inactivating effect Effects 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 108091008146 restriction endonucleases Proteins 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 108091006106 transcriptional activators Proteins 0.000 description 1
- 150000003751 zinc Chemical class 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023039486A JP7477675B2 (ja) | 2017-04-03 | 2023-03-14 | 組織選択的導入遺伝子発現 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762480998P | 2017-04-03 | 2017-04-03 | |
| US62/480,998 | 2017-04-03 | ||
| PCT/US2018/025940 WO2018187363A1 (en) | 2017-04-03 | 2018-04-03 | Tissue selective transgene expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023039486A Division JP7477675B2 (ja) | 2017-04-03 | 2023-03-14 | 組織選択的導入遺伝子発現 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020515292A JP2020515292A (ja) | 2020-05-28 |
| JP2020515292A5 true JP2020515292A5 (enExample) | 2021-05-13 |
| JP7246359B2 JP7246359B2 (ja) | 2023-03-27 |
Family
ID=63713399
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020502552A Active JP7246359B2 (ja) | 2017-04-03 | 2018-04-03 | 組織選択的導入遺伝子発現 |
| JP2023039486A Active JP7477675B2 (ja) | 2017-04-03 | 2023-03-14 | 組織選択的導入遺伝子発現 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023039486A Active JP7477675B2 (ja) | 2017-04-03 | 2023-03-14 | 組織選択的導入遺伝子発現 |
Country Status (20)
| Country | Link |
|---|---|
| US (6) | US10519465B2 (enExample) |
| EP (2) | EP3607073B1 (enExample) |
| JP (2) | JP7246359B2 (enExample) |
| KR (1) | KR102604159B1 (enExample) |
| CN (2) | CN110730823B (enExample) |
| AU (1) | AU2018250161B2 (enExample) |
| BR (1) | BR112019020777A2 (enExample) |
| CA (1) | CA3058189A1 (enExample) |
| CL (2) | CL2019002805A1 (enExample) |
| CO (1) | CO2019011450A2 (enExample) |
| EA (1) | EA201992358A1 (enExample) |
| ES (1) | ES2981970T3 (enExample) |
| IL (1) | IL269767A (enExample) |
| MA (1) | MA71412A (enExample) |
| MX (4) | MX2019011772A (enExample) |
| PL (1) | PL3607073T3 (enExample) |
| SA (1) | SA519410244B1 (enExample) |
| SG (1) | SG11201909203WA (enExample) |
| TW (3) | TWI848486B (enExample) |
| WO (1) | WO2018187363A1 (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| EP3390636B1 (en) | 2015-12-14 | 2021-05-19 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of dravet syndrome |
| CA3058189A1 (en) | 2017-04-03 | 2018-10-11 | Encoded Therapeutics, Inc. | Tissue selective transgene expression |
| EP3673080B1 (en) | 2017-08-25 | 2023-10-18 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| SG11202004926WA (en) | 2017-12-01 | 2020-06-29 | Encoded Therapeutics Inc | Engineered dna binding proteins |
| CA3096407A1 (en) * | 2018-04-09 | 2019-10-17 | Allen Institute | Rescuing voltage-gated sodium channel function in inhibitory neurons |
| JP2021523227A (ja) | 2018-05-04 | 2021-09-02 | ストーク セラピューティクス，インク． | コレステリルエステル蓄積症の処置のための方法及び組成物 |
| CN113166767B (zh) * | 2018-09-05 | 2025-03-28 | 马克思-德布鲁克-分子医学中心亥姆霍兹联合会 | 用于工程合成顺式调控dna的方法 |
| CN113966399A (zh) | 2018-09-26 | 2022-01-21 | 加州理工学院 | 用于靶向基因疗法的腺相关病毒组合物 |
| AU2019358806B2 (en) | 2018-10-08 | 2025-06-05 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in interneurons |
| AU2019375975B2 (en) * | 2018-11-05 | 2025-07-10 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons |
| CN113966400A (zh) * | 2019-02-05 | 2022-01-21 | 博德研究所 | 用于使神经元细胞兴奋性正常化和治疗德拉韦综合征的中间神经元特异性治疗剂 |
| CN113710693B (zh) * | 2019-02-25 | 2025-07-15 | 马萨诸塞大学 | Dna结合结构域反式激活因子及其用途 |
| CN113748209A (zh) | 2019-02-27 | 2021-12-03 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
| TW202102680A (zh) * | 2019-03-22 | 2021-01-16 | 美商編碼治療公司 | 多工處理調控元件以識別細胞類型專一性調控元件 |
| BR112021024055A2 (pt) * | 2019-05-29 | 2022-02-08 | Encoded Therapeutics Inc | Composições e métodos para a regulação seletiva de gene |
| AU2021270720A1 (en) | 2020-05-11 | 2022-12-08 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
| WO2022011390A1 (en) * | 2020-07-08 | 2022-01-13 | Baylor College Of Medicine | Gene therapy for stxbp1 encephalopathy |
| US12173304B2 (en) | 2020-07-08 | 2024-12-24 | Neuracle Genetics Inc. | Intron fragments |
| CA3182390A1 (en) * | 2020-07-09 | 2022-01-13 | Iain Robert THOMPSON | Method for treating alzheimer's disease by targeting mapt gene |
| AR123041A1 (es) * | 2020-07-24 | 2022-10-26 | Univ Massachusetts | Transactivadores del dominio de unión al adn y usos de los mismos |
| MX2024005171A (es) * | 2021-10-28 | 2024-05-13 | UCB Biopharma SRL | Constructos de acido nucleico, vectores viricos y particulas viricas. |
| WO2025212993A1 (en) * | 2024-04-05 | 2025-10-09 | Encoded Therapeutics, Inc. | Materials and methods for trangene expression in neural cells |
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-
2018
- 2018-04-03 CA CA3058189A patent/CA3058189A1/en active Pending
- 2018-04-03 ES ES18781685T patent/ES2981970T3/es active Active
- 2018-04-03 CN CN201880036950.6A patent/CN110730823B/zh active Active
- 2018-04-03 EP EP18781685.5A patent/EP3607073B1/en active Active
- 2018-04-03 TW TW111150116A patent/TWI848486B/zh active
- 2018-04-03 KR KR1020197032432A patent/KR102604159B1/ko active Active
- 2018-04-03 TW TW107111969A patent/TWI808079B/zh active
- 2018-04-03 JP JP2020502552A patent/JP7246359B2/ja active Active
- 2018-04-03 MA MA71412A patent/MA71412A/fr unknown
- 2018-04-03 EA EA201992358A patent/EA201992358A1/ru unknown
- 2018-04-03 WO PCT/US2018/025940 patent/WO2018187363A1/en not_active Ceased
- 2018-04-03 BR BR112019020777-5A patent/BR112019020777A2/pt unknown
- 2018-04-03 MX MX2019011772A patent/MX2019011772A/es unknown
- 2018-04-03 CN CN202410011187.9A patent/CN117821509A/zh active Pending
- 2018-04-03 EP EP24175675.8A patent/EP4403642A3/en active Pending
- 2018-04-03 PL PL18781685.5T patent/PL3607073T3/pl unknown
- 2018-04-03 SG SG11201909203W patent/SG11201909203WA/en unknown
- 2018-04-03 AU AU2018250161A patent/AU2018250161B2/en active Active
- 2018-04-03 TW TW111150117A patent/TW202315946A/zh unknown
- 2018-10-05 US US16/153,433 patent/US10519465B2/en active Active
- 2018-10-05 US US16/153,443 patent/US20190024121A1/en not_active Abandoned
- 2018-10-05 US US16/153,401 patent/US10287607B2/en active Active
- 2018-10-05 US US16/153,420 patent/US10287608B2/en active Active
-
2019
- 2019-09-30 MX MX2022015605A patent/MX2022015605A/es unknown
- 2019-09-30 MX MX2022015604A patent/MX2022015604A/es unknown
- 2019-09-30 MX MX2022015603A patent/MX2022015603A/es unknown
- 2019-10-01 CL CL2019002805A patent/CL2019002805A1/es unknown
- 2019-10-02 IL IL26976719A patent/IL269767A/en unknown
- 2019-10-03 SA SA519410244A patent/SA519410244B1/ar unknown
- 2019-10-16 CO CO2019011450A patent/CO2019011450A2/es unknown
- 2019-10-31 US US16/670,996 patent/US20200165628A1/en not_active Abandoned
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2022
- 2022-11-16 US US18/055,968 patent/US20230203531A1/en active Pending
-
2023
- 2023-01-30 CL CL2023000294A patent/CL2023000294A1/es unknown
- 2023-03-14 JP JP2023039486A patent/JP7477675B2/ja active Active
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