JP2019524160A5 - - Google Patents
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- Publication number
- JP2019524160A5 JP2019524160A5 JP2019529315A JP2019529315A JP2019524160A5 JP 2019524160 A5 JP2019524160 A5 JP 2019524160A5 JP 2019529315 A JP2019529315 A JP 2019529315A JP 2019529315 A JP2019529315 A JP 2019529315A JP 2019524160 A5 JP2019524160 A5 JP 2019524160A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- repeat dna
- administered
- dna sequence
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 32
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 10
- 208000023105 Huntington disease Diseases 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 102100032187 Androgen receptor Human genes 0.000 claims 2
- 101000775732 Homo sapiens Androgen receptor Proteins 0.000 claims 2
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 claims 2
- 208000006269 X-Linked Bulbo-Spinal Atrophy Diseases 0.000 claims 2
- 238000001727 in vivo Methods 0.000 claims 2
- 201000003624 spinocerebellar ataxia type 1 Diseases 0.000 claims 2
- 230000001225 therapeutic effect Effects 0.000 claims 2
- 241000272517 Anseriformes Species 0.000 claims 1
- 206010003694 Atrophy Diseases 0.000 claims 1
- 208000020925 Bipolar disease Diseases 0.000 claims 1
- 206010006187 Breast cancer Diseases 0.000 claims 1
- 208000026310 Breast neoplasm Diseases 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 101001026232 Homo sapiens Small conductance calcium-activated potassium channel protein 3 Proteins 0.000 claims 1
- 206010068871 Myotonic dystrophy Diseases 0.000 claims 1
- 102100037442 Small conductance calcium-activated potassium channel protein 3 Human genes 0.000 claims 1
- 101000693967 Trachemys scripta 67 kDa serum albumin Proteins 0.000 claims 1
- 230000037444 atrophy Effects 0.000 claims 1
- 201000004180 corneal endothelial dystrophy Diseases 0.000 claims 1
- 208000035928 fuchs endothelial 2 corneal dystrophy Diseases 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000007917 intracranial administration Methods 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 210000000463 red nucleus Anatomy 0.000 claims 1
- 201000000980 schizophrenia Diseases 0.000 claims 1
- 210000004291 uterus Anatomy 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662374072P | 2016-08-12 | 2016-08-12 | |
| US62/374,072 | 2016-08-12 | ||
| PCT/IB2017/054932 WO2018029660A1 (en) | 2016-08-12 | 2017-08-12 | Methods of treating diseases associated with repeat instability |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019524160A JP2019524160A (ja) | 2019-09-05 |
| JP2019524160A5 true JP2019524160A5 (enExample) | 2020-09-24 |
| JP7236707B2 JP7236707B2 (ja) | 2023-03-10 |
Family
ID=60009661
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019529315A Active JP7236707B2 (ja) | 2016-08-12 | 2017-08-12 | リピート不安定性と関連する疾患を治療する方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US11672786B2 (enExample) |
| EP (1) | EP3496713B1 (enExample) |
| JP (1) | JP7236707B2 (enExample) |
| CN (1) | CN110381943A (enExample) |
| CA (1) | CA3033590A1 (enExample) |
| WO (1) | WO2018029660A1 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7084611B2 (ja) * | 2018-04-06 | 2022-06-15 | 国立大学法人大阪大学 | 脊髄小脳変性症３１型抑制剤 |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
| JP5866283B2 (ja) * | 2009-07-28 | 2016-02-17 | サンガモ バイオサイエンシーズ， インコーポレイテッド | トリヌクレオチド反復疾患を治療するための方法および組成物 |
-
2017
- 2017-08-12 CA CA3033590A patent/CA3033590A1/en active Pending
- 2017-08-12 JP JP2019529315A patent/JP7236707B2/ja active Active
- 2017-08-12 EP EP17778331.3A patent/EP3496713B1/en active Active
- 2017-08-12 CN CN201780063252.0A patent/CN110381943A/zh active Pending
- 2017-08-12 WO PCT/IB2017/054932 patent/WO2018029660A1/en not_active Ceased
- 2017-08-12 US US16/325,066 patent/US11672786B2/en active Active
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