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 (enExample) | 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 (enExample) |
| EP (2) | EP3539980A4 (enExample) |
| JP (1) | JP7338937B2 (enExample) |
| KR (2) | KR102437228B1 (enExample) |
| CN (1) | CN110248957B (enExample) |
| AU (1) | AU2017358122B2 (enExample) |
| BR (1) | BR112019009725A2 (enExample) |
| CA (1) | CA3043148A1 (enExample) |
| RU (2) | RU2022105597A (enExample) |
| WO (1) | WO2018088694A2 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2767201C2 (ru) * | 2017-09-28 | 2022-03-16 | Тулджен Инкорпорейтед | Искусственная модификация генома для регуляции экспрессии гена |
| US11572574B2 (en) | 2017-09-28 | 2023-02-07 | Toolgen Incorporated | Artificial genome manipulation for gene expression regulation |
| JP7410154B2 (ja) * | 2018-09-12 | 2024-01-09 | インスティチュート フォー ベーシック サイエンス | 遺伝的に変異された細胞の死滅誘導組成物及び該組成物を用いた遺伝的に変異された細胞の死滅誘導方法 |
| 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 |
| CA2690815A1 (en) | 2007-06-12 | 2008-12-18 | Case Western Reserve University | Targeted cell death |
| CN110669758A (zh) | 2012-10-23 | 2020-01-10 | 基因工具股份有限公司 | 用于切割靶dna 的组合物及其用途 |
| RU2721275C2 (ru) | 2012-12-12 | 2020-05-18 | Те Брод Инститьют, Инк. | Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии |
| EP3011029B1 (en) | 2013-06-17 | 2019-12-11 | The Broad Institute, Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| EP3985124A1 (en) | 2013-12-26 | 2022-04-20 | The General Hospital Corporation | Multiplex guide rnas |
| WO2015188065A1 (en) | 2014-06-05 | 2015-12-10 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease design |
| CN113789317B (zh) | 2014-08-06 | 2024-02-23 | 基因工具股份有限公司 | 使用空肠弯曲杆菌crispr/cas系统衍生的rna引导的工程化核酸酶的基因编辑 |
| IL294014B2 (en) | 2015-10-23 | 2024-07-01 | Harvard College | Nucleobase editors and uses thereof |
-
2017
- 2017-09-28 WO PCT/KR2017/010897 patent/WO2018088694A2/ko not_active Ceased
- 2017-09-28 BR BR112019009725-2A patent/BR112019009725A2/pt unknown
- 2017-09-28 AU AU2017358122A patent/AU2017358122B2/en active Active
- 2017-09-28 EP EP17869218.2A patent/EP3539980A4/en not_active Withdrawn
- 2017-09-28 CA CA3043148A patent/CA3043148A1/en active Pending
- 2017-09-28 US US16/349,672 patent/US12331086B2/en active Active
- 2017-09-28 EP EP21166447.9A patent/EP3896162B1/en active Active
- 2017-09-28 KR KR1020170126636A patent/KR102437228B1/ko active Active
- 2017-09-28 RU RU2022105597A patent/RU2022105597A/ru unknown
- 2017-09-28 JP JP2019524226A patent/JP7338937B2/ja active Active
- 2017-09-28 CN CN201780083263.5A patent/CN110248957B/zh 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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