JPWO2020150338A5 - - Google Patents
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- Publication number
- JPWO2020150338A5 JPWO2020150338A5 JP2021540305A JP2021540305A JPWO2020150338A5 JP WO2020150338 A5 JPWO2020150338 A5 JP WO2020150338A5 JP 2021540305 A JP2021540305 A JP 2021540305A JP 2021540305 A JP2021540305 A JP 2021540305A JP WO2020150338 A5 JPWO2020150338 A5 JP WO2020150338A5
- Authority
- JP
- Japan
- Prior art keywords
- zfp
- composition
- polynucleotides
- striatum
- subject
- 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
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- 239000013608 rAAV vector Substances 0.000 claims description 19
- 102000040430 polynucleotide Human genes 0.000 claims description 16
- 108091033319 polynucleotide Proteins 0.000 claims description 16
- 239000002157 polynucleotide Substances 0.000 claims description 16
- 102000038627 Zinc finger transcription factors Human genes 0.000 claims description 14
- 108091007916 Zinc finger transcription factors Proteins 0.000 claims description 14
- 210000001577 neostriatum Anatomy 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 10
- 101710185494 Zinc finger protein Proteins 0.000 claims description 8
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 8
- 210000004556 brain Anatomy 0.000 claims description 6
- 210000004027 cell Anatomy 0.000 claims description 6
- 210000002569 neuron Anatomy 0.000 claims description 6
- 101150043003 Htt gene Proteins 0.000 claims description 4
- 208000023105 Huntington disease Diseases 0.000 claims description 4
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 4
- 239000013598 vector Substances 0.000 claims description 3
- 208000016285 Movement disease Diseases 0.000 claims description 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 13
- 238000000034 method Methods 0.000 description 7
- 230000002146 bilateral effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962792701P | 2019-01-15 | 2019-01-15 | |
| US62/792,701 | 2019-01-15 | ||
| PCT/US2020/013661 WO2020150338A1 (en) | 2019-01-15 | 2020-01-15 | Htt repressors and uses thereof |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022517988A JP2022517988A (ja) | 2022-03-11 |
| JP2022517988A5 JP2022517988A5 (https=) | 2023-01-25 |
| JPWO2020150338A5 true JPWO2020150338A5 (https=) | 2023-01-25 |
| JP7542912B2 JP7542912B2 (ja) | 2024-09-02 |
Family
ID=71614037
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021540305A Active JP7542912B2 (ja) | 2019-01-15 | 2020-01-15 | Ｈｔｔリプレッサーおよびその使用 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20220064237A1 (https=) |
| EP (1) | EP3911349A4 (https=) |
| JP (1) | JP7542912B2 (https=) |
| CN (1) | CN113301909B (https=) |
| WO (1) | WO2020150338A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019204457A1 (en) | 2018-04-18 | 2019-10-24 | Sangamo Therapeutics, Inc. | Zinc finger protein compositions for modulation of huntingtin (htt) |
| CN115487315B (zh) * | 2022-04-20 | 2023-08-04 | 暨南大学 | 治疗亨廷顿病的药物 |
| WO2025004001A1 (en) * | 2023-06-30 | 2025-01-02 | Takeda Pharmaceutical Company Limited | Htt repressors and uses thereof |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7888121B2 (en) * | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US7972854B2 (en) * | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| JP5266210B2 (ja) * | 2006-05-25 | 2013-08-21 | サンガモ バイオサイエンシズ インコーポレイテッド | 改変開裂ハーフドメイン |
| 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 |
| CN108285491B (zh) | 2012-02-29 | 2021-08-10 | 桑格摩生物科学股份有限公司 | 治疗亨廷顿氏病的方法和组合物 |
| WO2015070212A1 (en) * | 2013-11-11 | 2015-05-14 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
| CN106459894B (zh) * | 2014-03-18 | 2020-02-18 | 桑格摩生物科学股份有限公司 | 用于调控锌指蛋白表达的方法和组合物 |
| RU2691102C2 (ru) * | 2014-05-08 | 2019-06-11 | Сангамо Байосайенсиз, Инк. | Способы и композиции для лечения болезни хантингтона |
| WO2017053753A1 (en) | 2015-09-23 | 2017-03-30 | Sangamo Biosciences, Inc. | Htt repressors and uses thereof |
| GB2544270A (en) * | 2015-11-05 | 2017-05-17 | Fundació Centre De Regulació Genòmica | Nucleic acids, peptides and methods |
| WO2019204457A1 (en) * | 2018-04-18 | 2019-10-24 | Sangamo Therapeutics, Inc. | Zinc finger protein compositions for modulation of huntingtin (htt) |
-
2020
- 2020-01-15 EP EP20740948.3A patent/EP3911349A4/en active Pending
- 2020-01-15 CN CN202080009478.4A patent/CN113301909B/zh active Active
- 2020-01-15 JP JP2021540305A patent/JP7542912B2/ja active Active
- 2020-01-15 US US17/423,063 patent/US20220064237A1/en active Pending
- 2020-01-15 WO PCT/US2020/013661 patent/WO2020150338A1/en not_active Ceased
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