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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP3068905A4 (OSRAM) |
| JP (2) | JP2016537341A (OSRAM) |
| KR (1) | KR102431079B1 (OSRAM) |
| CN (1) | CN105934524A (OSRAM) |
| AU (1) | AU2014346424B2 (OSRAM) |
| BR (1) | BR112016010175A2 (OSRAM) |
| CA (1) | CA2929179A1 (OSRAM) |
| MX (1) | MX391904B (OSRAM) |
| NZ (1) | NZ719477A (OSRAM) |
| RU (1) | RU2693891C1 (OSRAM) |
| WO (1) | WO2015070212A1 (OSRAM) |
Families Citing this family (67)
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| 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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| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
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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 |
| CN111218447B (zh) | 2013-11-07 | 2024-10-11 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
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| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Cancer vaccine |
| CN110914310A (zh) | 2017-03-10 | 2020-03-24 | 哈佛大学的校长及成员们 | 胞嘧啶至鸟嘌呤碱基编辑器 |
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- 2014-11-11 BR BR112016010175A patent/BR112016010175A2/pt active Search and Examination
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- 2014-11-11 JP JP2016528904A patent/JP2016537341A/ja active Pending
- 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 RU RU2016123086A patent/RU2693891C1/ru active
- 2014-11-11 CA CA2929179A patent/CA2929179A1/en not_active Abandoned
- 2014-11-11 MX MX2016006106A patent/MX391904B/es unknown
- 2014-11-11 CN CN201480072869.5A patent/CN105934524A/zh active Pending
- 2014-11-11 EP EP14860665.0A patent/EP3068905A4/en not_active Withdrawn
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