JP2019537446A5 - - Google Patents
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- Publication number
- JP2019537446A5 JP2019537446A5 JP2019524226A JP2019524226A JP2019537446A5 JP 2019537446 A5 JP2019537446 A5 JP 2019537446A5 JP 2019524226 A JP2019524226 A JP 2019524226A JP 2019524226 A JP2019524226 A JP 2019524226A JP 2019537446 A5 JP2019537446 A5 JP 2019537446A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- sequence
- composition
- pharmaceutical composition
- domain
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims 27
- 108020004707 nucleic acids Proteins 0.000 claims 15
- 102000039446 nucleic acids Human genes 0.000 claims 15
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 12
- 239000000203 mixture Substances 0.000 claims 12
- 239000008194 pharmaceutical composition Substances 0.000 claims 12
- 101150038744 PMP22 gene Proteins 0.000 claims 11
- 238000010353 genetic engineering Methods 0.000 claims 11
- 230000000295 complement effect Effects 0.000 claims 9
- 108090000623 proteins and genes Proteins 0.000 claims 9
- 108091033409 CRISPR Proteins 0.000 claims 7
- 102000004169 proteins and genes Human genes 0.000 claims 6
- 102000004389 Ribonucleoproteins Human genes 0.000 claims 4
- 108010081734 Ribonucleoproteins Proteins 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 239000013603 viral vector Substances 0.000 claims 4
- 230000008685 targeting Effects 0.000 claims 3
- 241000589875 Campylobacter jejuni Species 0.000 claims 2
- 201000009009 Charcot-Marie-Tooth disease type 1A Diseases 0.000 claims 2
- 108091026890 Coding region Proteins 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 241000713666 Lentivirus Species 0.000 claims 2
- 241000700584 Simplexvirus Species 0.000 claims 2
- 241000193996 Streptococcus pyogenes Species 0.000 claims 2
- 241000700618 Vaccinia virus Species 0.000 claims 2
- 241000701161 unidentified adenovirus Species 0.000 claims 2
- 241001430294 unidentified retrovirus Species 0.000 claims 2
- 201000006868 Charcot-Marie-Tooth disease type 3 Diseases 0.000 claims 1
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 208000006411 Hereditary Sensory and Motor Neuropathy Diseases 0.000 claims 1
- 101001000631 Homo sapiens Peripheral myelin protein 22 Proteins 0.000 claims 1
- 101001082860 Homo sapiens Peroxisomal membrane protein 2 Proteins 0.000 claims 1
- 102100030564 Peroxisomal membrane protein 2 Human genes 0.000 claims 1
- 241000191967 Staphylococcus aureus Species 0.000 claims 1
- 241000194020 Streptococcus thermophilus Species 0.000 claims 1
- 239000008280 blood Substances 0.000 claims 1
- 210000004369 blood Anatomy 0.000 claims 1
- 230000004064 dysfunction Effects 0.000 claims 1
- 238000003208 gene overexpression Methods 0.000 claims 1
- 239000007943 implant Substances 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 201000001119 neuropathy Diseases 0.000 claims 1
- 230000007823 neuropathy Effects 0.000 claims 1
- 208000033808 peripheral neuropathy Diseases 0.000 claims 1
- 210000004116 schwann cell Anatomy 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662421516P | 2016-11-14 | 2016-11-14 | |
| US62/421,516 | 2016-11-14 | ||
| PCT/KR2017/010897 WO2018088694A2 (ko) | 2016-11-14 | 2017-09-28 | 인위적으로 조작된 sc 기능 조절 시스템 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019537446A JP2019537446A (ja) | 2019-12-26 |
| JP2019537446A5 true JP2019537446A5 (OSRAM) | 2020-11-12 |
| JP7338937B2 JP7338937B2 (ja) | 2023-09-05 |
Family
ID=62109586
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019524226A Active JP7338937B2 (ja) | 2016-11-14 | 2017-09-28 | 人工的に操作されたｓｃ機能制御システム |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12331086B2 (OSRAM) |
| EP (2) | EP3539980A4 (OSRAM) |
| JP (1) | JP7338937B2 (OSRAM) |
| KR (2) | KR102437228B1 (OSRAM) |
| CN (1) | CN110248957B (OSRAM) |
| AU (1) | AU2017358122B2 (OSRAM) |
| BR (1) | BR112019009725A2 (OSRAM) |
| CA (1) | CA3043148A1 (OSRAM) |
| RU (2) | RU2022105597A (OSRAM) |
| WO (1) | WO2018088694A2 (OSRAM) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11572574B2 (en) | 2017-09-28 | 2023-02-07 | Toolgen Incorporated | Artificial genome manipulation for gene expression regulation |
| BR112020006428A2 (pt) * | 2017-09-28 | 2020-09-24 | Toolgen Incorporated | manipulação de genoma artificial para regulação de expressão de genes |
| WO2020055187A1 (ko) * | 2018-09-12 | 2020-03-19 | 기초과학연구원 | 유전자가 변이된 세포의 사멸 유도 조성물 및 상기 조성물을 이용한 유전자가 변형된 세포 사멸 유도 방법 |
| EP3628735A1 (en) * | 2018-09-25 | 2020-04-01 | Centre National De La Recherche Scientifique | Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease |
| EP3798301A1 (en) * | 2019-09-25 | 2021-03-31 | KWS SAAT SE & Co. KGaA | Promoter repression |
| WO2021184021A1 (en) * | 2020-03-13 | 2021-09-16 | Codiak Biosciences, Inc. | Extracellular vesicle-aso constructs targeting pmp22 |
| CN114621922A (zh) * | 2022-02-15 | 2022-06-14 | 中山大学附属第五医院 | 一种大鼠海绵体神经来源施旺细胞的提取纯化和培养方法 |
| WO2025079278A1 (ja) * | 2023-10-10 | 2025-04-17 | 国立大学法人 東京医科歯科大学 | シャルコー・マリー・トゥース病の処置剤 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR100613038B1 (ko) * | 2004-07-07 | 2006-08-16 | 공주대학교 산학협력단 | 염색체 17ｐ11.2-ｐ12 지역의 중복을 제외한 유전자돌연변이를 발병 원인으로 하는 ＣＭＴ 질환의 진단방법및 진단키트 |
| WO2008154644A1 (en) | 2007-06-12 | 2008-12-18 | Case Western Reserve University | Targeted cell death |
| SG11201503059XA (en) | 2012-10-23 | 2015-06-29 | Toolgen Inc | Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof |
| BR112015013784A2 (pt) * | 2012-12-12 | 2017-07-11 | Massachusetts Inst Technology | aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas |
| JP6625971B2 (ja) * | 2013-06-17 | 2019-12-25 | ザ・ブロード・インスティテュート・インコーポレイテッド | 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化 |
| US20160237455A1 (en) * | 2013-09-27 | 2016-08-18 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| AU2014370416B2 (en) * | 2013-12-26 | 2021-03-11 | The General Hospital Corporation | Multiplex guide RNAs |
| EP3152319A4 (en) | 2014-06-05 | 2017-12-27 | Sangamo BioSciences, Inc. | Methods and compositions for nuclease design |
| AU2015299850B2 (en) * | 2014-08-06 | 2020-08-13 | Institute For Basic Science | Genome editing using Campylobacter jejuni CRISPR/CAS system-derived RGEN |
| IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and their uses |
-
2017
- 2017-09-28 RU RU2022105597A patent/RU2022105597A/ru unknown
- 2017-09-28 KR KR1020170126636A patent/KR102437228B1/ko active Active
- 2017-09-28 EP EP17869218.2A patent/EP3539980A4/en not_active Withdrawn
- 2017-09-28 CN CN201780083263.5A patent/CN110248957B/zh active Active
- 2017-09-28 EP EP21166447.9A patent/EP3896162B1/en active Active
- 2017-09-28 CA CA3043148A patent/CA3043148A1/en active Pending
- 2017-09-28 BR BR112019009725-2A patent/BR112019009725A2/pt unknown
- 2017-09-28 JP JP2019524226A patent/JP7338937B2/ja active Active
- 2017-09-28 WO PCT/KR2017/010897 patent/WO2018088694A2/ko not_active Ceased
- 2017-09-28 US US16/349,672 patent/US12331086B2/en active Active
- 2017-09-28 AU AU2017358122A patent/AU2017358122B2/en active Active
- 2017-09-28 RU RU2019118283A patent/RU2768043C2/ru active
-
2022
- 2022-08-24 KR KR1020220105836A patent/KR102621539B1/ko active Active
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