JP2017515819A5 - - Google Patents
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- JP2017515819A5 JP2017515819A5 JP2016566652A JP2016566652A JP2017515819A5 JP 2017515819 A5 JP2017515819 A5 JP 2017515819A5 JP 2016566652 A JP2016566652 A JP 2016566652A JP 2016566652 A JP2016566652 A JP 2016566652A JP 2017515819 A5 JP2017515819 A5 JP 2017515819A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- neuron
- neurons
- composition according
- mhtt
- 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.)
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- 210000002569 neuron Anatomy 0.000 claims description 26
- 208000023105 Huntington disease Diseases 0.000 claims description 18
- 239000003112 inhibitor Substances 0.000 claims description 5
- 108091033409 CRISPR Proteins 0.000 claims description 4
- 238000010354 CRISPR gene editing Methods 0.000 claims description 4
- 208000016285 Movement disease Diseases 0.000 claims description 4
- 101710163270 Nuclease Proteins 0.000 claims description 4
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 claims description 4
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 4
- 230000006907 apoptotic process Effects 0.000 claims description 4
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims description 4
- 108091033319 polynucleotide Proteins 0.000 claims description 4
- 102000040430 polynucleotide Human genes 0.000 claims description 4
- 239000002157 polynucleotide Substances 0.000 claims description 4
- 108090000623 proteins and genes Proteins 0.000 claims description 4
- 102000004169 proteins and genes Human genes 0.000 claims description 4
- 230000002776 aggregation Effects 0.000 claims description 3
- 238000004220 aggregation Methods 0.000 claims description 3
- 108700028369 Alleles Proteins 0.000 claims description 2
- 108091023040 Transcription factor Proteins 0.000 claims description 2
- 102000040945 Transcription factor Human genes 0.000 claims description 2
- 108091007916 Zinc finger transcription factors Proteins 0.000 claims description 2
- 102000038627 Zinc finger transcription factors Human genes 0.000 claims description 2
- 230000015572 biosynthetic process Effects 0.000 claims description 2
- 230000001413 cellular effect Effects 0.000 claims description 2
- 239000003153 chemical reaction reagent Substances 0.000 claims description 2
- 230000000694 effects Effects 0.000 claims description 2
- 230000037149 energy metabolism Effects 0.000 claims description 2
- 230000003834 intracellular effect Effects 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 238000012986 modification Methods 0.000 claims description 2
- 230000004048 modification Effects 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 238000013518 transcription Methods 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 claims description 2
- 230000004075 alteration Effects 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 19
- 230000006735 deficit Effects 0.000 description 2
- 230000035945 sensitivity Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461990521P | 2014-05-08 | 2014-05-08 | |
| US61/990,521 | 2014-05-08 | ||
| US201462051724P | 2014-09-17 | 2014-09-17 | |
| US62/051,724 | 2014-09-17 | ||
| PCT/US2015/029748 WO2015171932A1 (en) | 2014-05-08 | 2015-05-07 | Methods and compositions for treating huntington's disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017515819A JP2017515819A (ja) | 2017-06-15 |
| JP2017515819A5 true JP2017515819A5 (enExample) | 2018-04-05 |
Family
ID=54393014
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016566652A Pending JP2017515819A (ja) | 2014-05-08 | 2015-05-07 | 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 (11)
| Country | Link |
|---|---|
| US (1) | US11110154B2 (enExample) |
| EP (1) | EP3139966B1 (enExample) |
| JP (1) | JP2017515819A (enExample) |
| KR (1) | KR102380324B1 (enExample) |
| CN (1) | CN106413760B (enExample) |
| AU (1) | AU2015255877B2 (enExample) |
| BR (1) | BR112016025849A2 (enExample) |
| CA (1) | CA2947035A1 (enExample) |
| MX (1) | MX378789B (enExample) |
| RU (1) | RU2691102C2 (enExample) |
| WO (1) | WO2015171932A1 (enExample) |
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2015
- 2015-05-07 JP JP2016566652A patent/JP2017515819A/ja active Pending
- 2015-05-07 WO PCT/US2015/029748 patent/WO2015171932A1/en not_active Ceased
- 2015-05-07 RU RU2016147932A patent/RU2691102C2/ru active
- 2015-05-07 AU AU2015255877A patent/AU2015255877B2/en active Active
- 2015-05-07 CA CA2947035A patent/CA2947035A1/en active Pending
- 2015-05-07 BR BR112016025849A patent/BR112016025849A2/pt not_active Application Discontinuation
- 2015-05-07 MX MX2016014565A patent/MX378789B/es unknown
- 2015-05-07 EP EP15789214.2A patent/EP3139966B1/en active Active
- 2015-05-07 KR KR1020167031778A patent/KR102380324B1/ko active Active
- 2015-05-07 CN CN201580024010.1A patent/CN106413760B/zh active Active
- 2015-05-07 US US14/706,747 patent/US11110154B2/en active Active
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