JP2017532035A5 - - Google Patents
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- Publication number
- JP2017532035A5 JP2017532035A5 JP2017518840A JP2017518840A JP2017532035A5 JP 2017532035 A5 JP2017532035 A5 JP 2017532035A5 JP 2017518840 A JP2017518840 A JP 2017518840A JP 2017518840 A JP2017518840 A JP 2017518840A JP 2017532035 A5 JP2017532035 A5 JP 2017532035A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- vector
- polynucleotide
- endonuclease
- hcsf1
- 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 56
- 108091033319 polynucleotide Proteins 0.000 claims description 45
- 102000040430 polynucleotide Human genes 0.000 claims description 45
- 239000002157 polynucleotide Substances 0.000 claims description 45
- 108010042407 Endonucleases Proteins 0.000 claims description 26
- 102000004533 Endonucleases Human genes 0.000 claims description 26
- 101000916628 Homo sapiens Macrophage colony-stimulating factor 1 Proteins 0.000 claims description 26
- 210000003594 spinal ganglia Anatomy 0.000 claims description 22
- 210000000427 trigeminal ganglion Anatomy 0.000 claims description 20
- 102000053925 human CSF1 Human genes 0.000 claims description 18
- 238000002347 injection Methods 0.000 claims description 17
- 239000007924 injection Substances 0.000 claims description 17
- 239000002773 nucleotide Substances 0.000 claims description 15
- 125000003729 nucleotide group Chemical group 0.000 claims description 15
- 101710163270 Nuclease Proteins 0.000 claims description 12
- 238000000034 method Methods 0.000 claims description 12
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 10
- 108091033409 CRISPR Proteins 0.000 claims description 10
- 102000014450 RNA Polymerase III Human genes 0.000 claims description 8
- 108010078067 RNA Polymerase III Proteins 0.000 claims description 8
- 230000001939 inductive effect Effects 0.000 claims description 8
- 241000700584 Simplexvirus Species 0.000 claims description 6
- 230000002401 inhibitory effect Effects 0.000 claims description 6
- 238000007913 intrathecal administration Methods 0.000 claims description 5
- 230000001177 retroviral effect Effects 0.000 claims description 5
- 101000654356 Homo sapiens Sodium channel protein type 10 subunit alpha Proteins 0.000 claims description 4
- 101000640020 Homo sapiens Sodium channel protein type 11 subunit alpha Proteins 0.000 claims description 4
- 101000654386 Homo sapiens Sodium channel protein type 9 subunit alpha Proteins 0.000 claims description 4
- 102100031374 Sodium channel protein type 10 subunit alpha Human genes 0.000 claims description 4
- 102100033974 Sodium channel protein type 11 subunit alpha Human genes 0.000 claims description 4
- 102100031367 Sodium channel protein type 9 subunit alpha Human genes 0.000 claims description 4
- 238000010459 TALEN Methods 0.000 claims description 4
- 102000003566 TRPV1 Human genes 0.000 claims description 4
- 101150016206 Trpv1 gene Proteins 0.000 claims description 4
- 108091026822 U6 spliceosomal RNA Proteins 0.000 claims description 4
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 4
- 230000014509 gene expression Effects 0.000 claims description 4
- 238000001802 infusion Methods 0.000 claims description 4
- 238000000185 intracerebroventricular administration Methods 0.000 claims description 4
- 208000004296 neuralgia Diseases 0.000 claims description 4
- 208000021722 neuropathic pain Diseases 0.000 claims description 4
- 102000039446 nucleic acids Human genes 0.000 claims description 4
- 108020004707 nucleic acids Proteins 0.000 claims description 4
- 150000007523 nucleic acids Chemical class 0.000 claims description 4
- 239000004055 small Interfering RNA Substances 0.000 claims description 4
- 238000010254 subcutaneous injection Methods 0.000 claims description 4
- 239000007929 subcutaneous injection Substances 0.000 claims description 4
- 239000013603 viral vector Substances 0.000 claims description 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
- 108090000994 Catalytic RNA Proteins 0.000 claims description 2
- 102000053642 Catalytic RNA Human genes 0.000 claims description 2
- 108010051219 Cre recombinase Proteins 0.000 claims description 2
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 2
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 108700035841 GIY-YIG endonucleases Proteins 0.000 claims description 2
- 102000052907 GIY-YIG endonucleases Human genes 0.000 claims description 2
- 108020005004 Guide RNA Proteins 0.000 claims description 2
- 108050008753 HNH endonucleases Proteins 0.000 claims description 2
- 102000000310 HNH endonucleases Human genes 0.000 claims description 2
- MAJYPBAJPNUFPV-BQBZGAKWSA-N His-Cys Chemical compound SC[C@@H](C(O)=O)NC(=O)[C@@H](N)CC1=CN=CN1 MAJYPBAJPNUFPV-BQBZGAKWSA-N 0.000 claims description 2
- 101000830950 Homo sapiens Three prime repair exonuclease 2 Proteins 0.000 claims description 2
- 206010020751 Hypersensitivity Diseases 0.000 claims description 2
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims description 2
- 241000588650 Neisseria meningitidis Species 0.000 claims description 2
- 208000028389 Nerve injury Diseases 0.000 claims description 2
- 108091007412 Piwi-interacting RNA Proteins 0.000 claims description 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 2
- 108020004459 Small interfering RNA Proteins 0.000 claims description 2
- 241000193996 Streptococcus pyogenes Species 0.000 claims description 2
- 241000194020 Streptococcus thermophilus Species 0.000 claims description 2
- 108091027544 Subgenomic mRNA Proteins 0.000 claims description 2
- 239000004098 Tetracycline Substances 0.000 claims description 2
- 102100024872 Three prime repair exonuclease 2 Human genes 0.000 claims description 2
- 108091036066 Three prime untranslated region Proteins 0.000 claims description 2
- 108091028113 Trans-activating crRNA Proteins 0.000 claims description 2
- 241000589892 Treponema denticola Species 0.000 claims description 2
- 208000026935 allergic disease Diseases 0.000 claims description 2
- 230000036592 analgesia Effects 0.000 claims description 2
- 108010050663 endodeoxyribonuclease CreI Proteins 0.000 claims description 2
- 230000009610 hypersensitivity Effects 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 108091070501 miRNA Proteins 0.000 claims description 2
- 239000002679 microRNA Substances 0.000 claims description 2
- 230000006724 microglial activation Effects 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 210000005036 nerve Anatomy 0.000 claims description 2
- 230000008764 nerve damage Effects 0.000 claims description 2
- 210000000578 peripheral nerve Anatomy 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 230000001105 regulatory effect Effects 0.000 claims description 2
- 108091092562 ribozyme Proteins 0.000 claims description 2
- 210000003497 sciatic nerve Anatomy 0.000 claims description 2
- 239000002924 silencing RNA Substances 0.000 claims description 2
- 230000008685 targeting Effects 0.000 claims description 2
- 229960002180 tetracycline Drugs 0.000 claims description 2
- 229930101283 tetracycline Natural products 0.000 claims description 2
- 235000019364 tetracycline Nutrition 0.000 claims description 2
- 150000003522 tetracyclines Chemical class 0.000 claims description 2
- 238000013518 transcription Methods 0.000 claims description 2
- 230000035897 transcription Effects 0.000 claims description 2
- 210000003901 trigeminal nerve Anatomy 0.000 claims description 2
- 230000009278 visceral effect Effects 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims 10
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 210000004556 brain Anatomy 0.000 description 1
- 238000007917 intracranial administration Methods 0.000 description 1
- 241000701161 unidentified adenovirus Species 0.000 description 1
- 241001430294 unidentified retrovirus Species 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462062047P | 2014-10-09 | 2014-10-09 | |
| US62/062,047 | 2014-10-09 | ||
| PCT/US2015/054704 WO2016057800A1 (en) | 2014-10-09 | 2015-10-08 | Targeted disruption of a csf1-dap12 pathway member gene for the treatment of neuropathic pain |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020107553A Division JP2020191877A (ja) | 2014-10-09 | 2020-06-23 | 神経障害性疼痛を治療するためのｃｓｆ１−ｄａｐ１２経路のメンバー遺伝子の標的破壊 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017532035A JP2017532035A (ja) | 2017-11-02 |
| JP2017532035A5 true JP2017532035A5 (https=) | 2018-11-15 |
| JP6723230B2 JP6723230B2 (ja) | 2020-07-15 |
Family
ID=55653779
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017518840A Expired - Fee Related JP6723230B2 (ja) | 2014-10-09 | 2015-10-08 | 神経障害性疼痛を治療するためのｃｓｆ１−ｄａｐ１２経路のメンバー遺伝子の標的破壊 |
| JP2020107553A Pending JP2020191877A (ja) | 2014-10-09 | 2020-06-23 | 神経障害性疼痛を治療するためのｃｓｆ１−ｄａｐ１２経路のメンバー遺伝子の標的破壊 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020107553A Pending JP2020191877A (ja) | 2014-10-09 | 2020-06-23 | 神経障害性疼痛を治療するためのｃｓｆ１−ｄａｐ１２経路のメンバー遺伝子の標的破壊 |
Country Status (5)
| Country | Link |
|---|---|
| US (2) | US20170354745A1 (https=) |
| EP (1) | EP3204050A4 (https=) |
| JP (2) | JP6723230B2 (https=) |
| CA (1) | CA2963940A1 (https=) |
| WO (1) | WO2016057800A1 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10900034B2 (en) | 2014-12-03 | 2021-01-26 | Agilent Technologies, Inc. | Guide RNA with chemical modifications |
| US10767175B2 (en) | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
| AU2017292173B2 (en) * | 2016-07-06 | 2022-01-13 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of pain related disorders |
| JP7305534B2 (ja) | 2016-07-06 | 2023-07-10 | バーテックス ファーマシューティカルズ インコーポレイテッド | 疼痛関連障害を処置するための物質及び方法 |
| US20200270604A1 (en) | 2017-09-19 | 2020-08-27 | Tropic Biosciences UK Limited | Modifying the specificity of non-coding rna molecules for silencing gene expression in eukaryotic cells |
| CN107557394A (zh) * | 2017-09-29 | 2018-01-09 | 南京鼓楼医院 | 降低CRISPR/Cas9介导的胚胎基因编辑脱靶率的方法 |
| GB201903519D0 (en) * | 2019-03-14 | 2019-05-01 | Tropic Biosciences Uk Ltd | Introducing silencing activity to dysfunctional rna molecules and modifying their specificity against a gene of interest |
| CN114729363A (zh) * | 2019-11-15 | 2022-07-08 | 诺格拉制药有限公司 | Il-34反义剂及其使用方法 |
| GB201918879D0 (en) * | 2019-12-19 | 2020-02-05 | Ucl Business Ltd | Treatment of chronic pain |
| WO2022079082A1 (en) | 2020-10-15 | 2022-04-21 | F. Hoffmann-La Roche Ag | Nucleic acid constructs for simultaneous gene activation |
| EP4229205B1 (en) | 2020-10-15 | 2025-05-14 | F. Hoffmann-La Roche AG | Nucleic acid constructs for va rna transcription |
| AU2022276078A1 (en) | 2021-05-17 | 2024-01-04 | Nogra Pharma Limited | Il-34 antisense agents and methods of using same |
| JP2024534945A (ja) | 2021-09-10 | 2024-09-26 | アジレント・テクノロジーズ・インク | 化学修飾を有するプライム編集のためのガイドｒｎａ |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009019528A1 (en) * | 2007-08-03 | 2009-02-12 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human interleukin-2 receptor gamma chain gene and uses thereof |
| US9173928B2 (en) * | 2009-03-26 | 2015-11-03 | Yoh Matsumoto | DNA vaccine for Alzheimer's disease |
| WO2011135396A1 (en) * | 2010-04-30 | 2011-11-03 | Cellectis | Method for modulating double-strand break-induced homologous recombination |
| JP2014520873A (ja) * | 2011-07-18 | 2014-08-25 | ザ ユニバーシティ オブ メルボルン | ｃ−Ｆｍｓアンタゴニストの使用 |
| DK2931897T3 (en) * | 2012-12-12 | 2018-02-05 | Broad Inst Inc | CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS |
-
2015
- 2015-10-08 WO PCT/US2015/054704 patent/WO2016057800A1/en not_active Ceased
- 2015-10-08 CA CA2963940A patent/CA2963940A1/en not_active Abandoned
- 2015-10-08 EP EP15848341.2A patent/EP3204050A4/en not_active Withdrawn
- 2015-10-08 JP JP2017518840A patent/JP6723230B2/ja not_active Expired - Fee Related
- 2015-10-08 US US15/516,872 patent/US20170354745A1/en not_active Abandoned
-
2019
- 2019-10-08 US US16/596,164 patent/US20200030459A1/en not_active Abandoned
-
2020
- 2020-06-23 JP JP2020107553A patent/JP2020191877A/ja active Pending
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