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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- 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) | US10287607B2 (enExample) |
| EP (2) | EP4403642A3 (enExample) |
| JP (2) | JP7246359B2 (enExample) |
| KR (1) | KR102604159B1 (enExample) |
| CN (2) | CN117821509A (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 (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CA2963288A1 (en) | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| MA71412A (fr) | 2017-04-03 | 2025-04-30 | Encoded Therapeutics, Inc. | Expression de transgène sélective d'un tissu |
| LT3673080T (lt) | 2017-08-25 | 2023-12-27 | Stoke Therapeutics, Inc. | Priešprasminiai oligomerai, skirti būklėms ir ligoms gydyti |
| JP7401432B2 (ja) | 2017-12-01 | 2023-12-19 | エンコーデッド セラピューティクス， インコーポレイテッド | 操作されたｄｎａ結合タンパク質 |
| WO2019199867A1 (en) * | 2018-04-09 | 2019-10-17 | Allen Institute | Rescuing voltage-gated sodium channel function in inhibitory neurons |
| BR112020022512A2 (pt) | 2018-05-04 | 2021-05-04 | Stoke Therapeutics, Inc. | métodos e composições para tratamento de doença de armazenamento de éster de colesteril |
| 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 |
| CA3118689A1 (en) * | 2018-11-05 | 2020-05-14 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons |
| US20220195457A1 (en) * | 2019-02-05 | 2022-06-23 | The Broad Institute, Inc. | Interneuron-specific therapeutics for normalizing neuronal cell excitability and treating dravet syndrome |
| JP7624728B2 (ja) * | 2019-02-25 | 2025-01-31 | ユニバーシティ オブ マサチューセッツ | Ｄｎａ結合ドメイントランスアクチベーター及びその使用 |
| EP3942055A4 (en) * | 2019-03-22 | 2022-12-28 | Encoded Therapeutics, Inc. | MULTIPLEXING OF REGULATORY ELEMENTS TO IDENTIFY CELL TYPE SPECIFIC REGULATORY ELEMENTS |
| CA3141900C (en) * | 2019-05-29 | 2023-06-06 | Encoded Therapeutics, Inc. | Compositions and methods for selective gene regulation |
| EP4150092A4 (en) | 2020-05-11 | 2024-11-06 | Stoke Therapeutics, Inc. | OPA1 ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASES AND CONDITIONS |
| 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 |
| CN115867652A (zh) * | 2020-07-09 | 2023-03-28 | 摩大力斯医疗株式会社 | 通过靶向mapt基因治疗阿尔茨海默病的方法 |
| US20230279405A1 (en) * | 2020-07-24 | 2023-09-07 | University Of Massachusetts | Dna-binding domain transactivators and uses thereof |
| IL312340A (en) * | 2021-10-28 | 2024-06-01 | UCB Biopharma SRL | Nucleic acid constructs, viral vectors and viral particles |
| WO2025212993A1 (en) * | 2024-04-05 | 2025-10-09 | Encoded Therapeutics, Inc. | Materials and methods for trangene expression in neural cells |
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| US7405A (en) * | 1850-05-28 | John weidman | ||
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| US6436708B1 (en) | 1997-04-17 | 2002-08-20 | Paola Leone | Delivery system for gene therapy to the brain |
| CA2205076A1 (en) | 1997-05-14 | 1998-11-14 | Jim Hu | Episomal expression cassettes for gene therapy |
| AU746454B2 (en) | 1998-03-02 | 2002-05-02 | Massachusetts Institute Of Technology | Poly zinc finger proteins with improved linkers |
| US6303370B1 (en) | 1998-03-24 | 2001-10-16 | Mayo Foundation For Medical Education And Research | Tissue-specific regulatory elements |
| CA2246005A1 (en) | 1998-10-01 | 2000-04-01 | Hsc Research And Development Limited Partnership | Hybrid genes for gene therapy in erythroid cells |
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| US6649371B1 (en) | 1999-06-11 | 2003-11-18 | Neurosearch A/S | Potassium channel KCNQ5 and sequences encoding the same |
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| AU2003219933A1 (en) | 2002-02-25 | 2003-09-09 | Vanderbilt University | Expression system for human brain-specific voltage-gated sodium channel, type 1 |
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| BR112015021884A8 (pt) | 2013-03-15 | 2019-11-26 | Childrens Hospital Philadelphia | vetores de plasmídeo recombinantes, partícula viral avv ou pluralidade de partículas virais, seus métodos de produção e seus usos, e composição farmacêutica |
| HK1222093A1 (zh) | 2013-05-15 | 2017-06-23 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
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| US11020443B2 (en) | 2015-04-23 | 2021-06-01 | University Of Massachusetts | Modulation of AAV vector transgene expression |
| CN104846015B (zh) | 2015-05-27 | 2018-03-27 | 深圳先进技术研究院 | 特异性兴奋伏隔核中的gaba能神经元的组合物及其在改善精神分裂症异样行为中的应用 |
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-
2018
- 2018-04-03 MA MA71412A patent/MA71412A/fr unknown
- 2018-04-03 KR KR1020197032432A patent/KR102604159B1/ko active Active
- 2018-04-03 BR BR112019020777A patent/BR112019020777A2/pt unknown
- 2018-04-03 JP JP2020502552A patent/JP7246359B2/ja active Active
- 2018-04-03 PL PL18781685.5T patent/PL3607073T3/pl unknown
- 2018-04-03 MX MX2019011772A patent/MX2019011772A/es unknown
- 2018-04-03 AU AU2018250161A patent/AU2018250161B2/en active Active
- 2018-04-03 EA EA201992358A patent/EA201992358A1/ru unknown
- 2018-04-03 EP EP24175675.8A patent/EP4403642A3/en active Pending
- 2018-04-03 EP EP18781685.5A patent/EP3607073B1/en active Active
- 2018-04-03 CN CN202410011187.9A patent/CN117821509A/zh active Pending
- 2018-04-03 CA CA3058189A patent/CA3058189A1/en active Pending
- 2018-04-03 CN CN201880036950.6A patent/CN110730823B/zh active Active
- 2018-04-03 TW TW111150116A patent/TWI848486B/zh active
- 2018-04-03 TW TW107111969A patent/TWI808079B/zh active
- 2018-04-03 ES ES18781685T patent/ES2981970T3/es active Active
- 2018-04-03 SG SG11201909203W patent/SG11201909203WA/en unknown
- 2018-04-03 TW TW111150117A patent/TW202315946A/zh unknown
- 2018-04-03 WO PCT/US2018/025940 patent/WO2018187363A1/en not_active Ceased
- 2018-10-05 US US16/153,401 patent/US10287607B2/en active Active
- 2018-10-05 US US16/153,420 patent/US10287608B2/en active Active
- 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
-
2019
- 2019-09-30 MX MX2022015603A patent/MX2022015603A/es unknown
- 2019-09-30 MX MX2022015604A patent/MX2022015604A/es unknown
- 2019-09-30 MX MX2022015605A patent/MX2022015605A/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
-
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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