JP2016537341A5 - - Google Patents
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- JP2016537341A5 JP2016537341A5 JP2016528904A JP2016528904A JP2016537341A5 JP 2016537341 A5 JP2016537341 A5 JP 2016537341A5 JP 2016528904 A JP2016528904 A JP 2016528904A JP 2016528904 A JP2016528904 A JP 2016528904A JP 2016537341 A5 JP2016537341 A5 JP 2016537341A5
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- 108090000623 proteins and genes Proteins 0.000 claims description 12
- 102100020802 D(1A) dopamine receptor Human genes 0.000 claims description 6
- 102100020756 D(2) dopamine receptor Human genes 0.000 claims description 6
- 230000004568 DNA-binding Effects 0.000 claims description 6
- 102000012749 Dopamine and cAMP-Regulated Phosphoprotein 32 Human genes 0.000 claims description 6
- 108010090047 Dopamine and cAMP-Regulated Phosphoprotein 32 Proteins 0.000 claims description 6
- 101000931925 Homo sapiens D(1A) dopamine receptor Proteins 0.000 claims description 6
- 101000931901 Homo sapiens D(2) dopamine receptor Proteins 0.000 claims description 6
- 108020004707 nucleic acids Proteins 0.000 claims description 6
- 102000039446 nucleic acids Human genes 0.000 claims description 6
- 150000007523 nucleic acids Chemical class 0.000 claims description 6
- 230000000754 repressing effect Effects 0.000 claims description 5
- 239000013603 viral vector Substances 0.000 claims description 5
- 102000016252 Huntingtin Human genes 0.000 claims description 4
- 108050004784 Huntingtin Proteins 0.000 claims description 4
- 210000003169 central nervous system Anatomy 0.000 claims description 4
- 210000002569 neuron Anatomy 0.000 claims description 4
- 208000023105 Huntington disease Diseases 0.000 claims description 3
- 239000013598 vector Substances 0.000 claims description 3
- 108091033409 CRISPR Proteins 0.000 claims description 2
- 238000010354 CRISPR gene editing Methods 0.000 claims description 2
- 108020004414 DNA Proteins 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 101710163270 Nuclease Proteins 0.000 claims description 2
- 108091005461 Nucleic proteins Proteins 0.000 claims description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 claims description 2
- 101710185494 Zinc finger protein Proteins 0.000 claims description 2
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 2
- 230000002068 genetic effect Effects 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 150000003384 small molecules Chemical group 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 15
- 101001072037 Homo sapiens cAMP and cAMP-inhibited cGMP 3',5'-cyclic phosphodiesterase 10A Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 102100036377 cAMP and cAMP-inhibited cGMP 3',5'-cyclic phosphodiesterase 10A Human genes 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 230000030279 gene silencing Effects 0.000 claims 1
- 238000012226 gene silencing method Methods 0.000 claims 1
- 230000037426 transcriptional repression Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 17
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 210000001577 neostriatum Anatomy 0.000 description 1
- 238000013518 transcription Methods 0.000 description 1
- 230000035897 transcription Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361902704P | 2013-11-11 | 2013-11-11 | |
| US61/902,704 | 2013-11-11 | ||
| PCT/US2014/064987 WO2015070212A1 (en) | 2013-11-11 | 2014-11-11 | Methods and compositions for treating huntington's disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019067000A Division JP2019116502A (ja) | 2013-11-11 | 2019-03-29 | ハンチントン病を処置するための方法および組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016537341A JP2016537341A (ja) | 2016-12-01 |
| JP2016537341A5 true JP2016537341A5 (enExample) | 2017-10-12 |
Family
ID=53042231
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016528904A Pending JP2016537341A (ja) | 2013-11-11 | 2014-11-11 | ハンチントン病を処置するための方法および組成物 |
| JP2019067000A Pending JP2019116502A (ja) | 2013-11-11 | 2019-03-29 | ハンチントン病を処置するための方法および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019067000A Pending JP2019116502A (ja) | 2013-11-11 | 2019-03-29 | ハンチントン病を処置するための方法および組成物 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US10369201B2 (enExample) |
| EP (1) | EP3068905A4 (enExample) |
| JP (2) | JP2016537341A (enExample) |
| KR (1) | KR102431079B1 (enExample) |
| CN (1) | CN105934524A (enExample) |
| AU (1) | AU2014346424B2 (enExample) |
| BR (1) | BR112016010175A2 (enExample) |
| CA (1) | CA2929179A1 (enExample) |
| MX (1) | MX391904B (enExample) |
| NZ (1) | NZ719477A (enExample) |
| RU (1) | RU2693891C1 (enExample) |
| WO (1) | WO2015070212A1 (enExample) |
Families Citing this family (67)
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| IN2012DN00720A (enExample) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| MX347361B (es) | 2011-07-19 | 2017-04-12 | Wave Life Sciences Ltd | Metodos para la sintesis de acidos nucleicos funcionalizados. |
| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ヌクレアーゼ切断特異性の評価および改善 |
| EP2872147B1 (en) | 2012-07-13 | 2022-12-21 | Wave Life Sciences Ltd. | Method for making chiral oligonucleotides |
| EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| AU2013287630B2 (en) | 2012-07-13 | 2017-05-25 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
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| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| PT3066201T (pt) | 2013-11-07 | 2018-06-04 | Massachusetts Inst Technology | Métodos e composições relacionadas com crispr com arng reguladores |
| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
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| WO2019236082A1 (en) * | 2018-06-06 | 2019-12-12 | The Regents Of The University Of California | Neural stem cell compositions and methods to treat neurodegenerative disorders |
| CN109112162B (zh) * | 2018-08-30 | 2021-10-15 | 西南大学 | 利用CRISPR/Cas9技术构建的亨廷顿病原位敲入小鼠模型及构建方法 |
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-
2014
- 2014-11-11 JP JP2016528904A patent/JP2016537341A/ja active Pending
- 2014-11-11 BR BR112016010175A patent/BR112016010175A2/pt active Search and Examination
- 2014-11-11 EP EP14860665.0A patent/EP3068905A4/en not_active Withdrawn
- 2014-11-11 CN CN201480072869.5A patent/CN105934524A/zh active Pending
- 2014-11-11 NZ NZ719477A patent/NZ719477A/en unknown
- 2014-11-11 MX MX2016006106A patent/MX391904B/es unknown
- 2014-11-11 CA CA2929179A patent/CA2929179A1/en not_active Abandoned
- 2014-11-11 US US15/036,020 patent/US10369201B2/en active Active
- 2014-11-11 WO PCT/US2014/064987 patent/WO2015070212A1/en not_active Ceased
- 2014-11-11 AU AU2014346424A patent/AU2014346424B2/en active Active
- 2014-11-11 KR KR1020167015301A patent/KR102431079B1/ko active Active
- 2014-11-11 RU RU2016123086A patent/RU2693891C1/ru active
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2019
- 2019-03-29 JP JP2019067000A patent/JP2019116502A/ja active Pending
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