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
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- Prior art keywords
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- neuron
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- mhtt
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- 210000002569 neurons Anatomy 0.000 claims description 26
- 201000001971 Huntington's disease Diseases 0.000 claims description 18
- 101700080605 NUC1 Proteins 0.000 claims description 6
- 230000002401 inhibitory effect Effects 0.000 claims description 6
- 101700006494 nucA Proteins 0.000 claims description 6
- 239000003112 inhibitor Substances 0.000 claims description 5
- 206010059512 Apoptosis Diseases 0.000 claims description 4
- 229920000033 CRISPR Polymers 0.000 claims description 4
- 238000010354 CRISPR gene editing Methods 0.000 claims description 4
- 210000001175 Cerebrospinal Fluid Anatomy 0.000 claims description 4
- 230000006907 apoptotic process Effects 0.000 claims description 4
- 201000009457 movement disease Diseases 0.000 claims description 4
- 229920000023 polynucleotide Polymers 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
- ZKHQWZAMYRWXGA-KQYNXXCUSA-N Adenosine triphosphate Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)[C@@H](O)[C@H]1O ZKHQWZAMYRWXGA-KQYNXXCUSA-N 0.000 claims description 2
- 108020004999 Messenger RNA Proteins 0.000 claims description 2
- 102000038033 Zinc finger transcription factors Human genes 0.000 claims description 2
- 108091007478 Zinc finger transcription factors Proteins 0.000 claims description 2
- 230000015572 biosynthetic process Effects 0.000 claims description 2
- 230000001413 cellular 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
- 238000005755 formation reaction Methods 0.000 claims description 2
- 230000003834 intracellular Effects 0.000 claims description 2
- 229920002106 messenger RNA Polymers 0.000 claims description 2
- 230000004048 modification Effects 0.000 claims description 2
- 238000006011 modification reaction Methods 0.000 claims description 2
- 102000003995 transcription factors Human genes 0.000 claims description 2
- 108090000464 transcription factors Proteins 0.000 claims description 2
- 230000003612 virological Effects 0.000 claims description 2
- HCHKCACWOHOZIP-UHFFFAOYSA-N zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims description 2
- 239000011701 zinc Substances 0.000 claims description 2
- 229910052725 zinc Inorganic materials 0.000 claims description 2
- 230000004075 alteration Effects 0.000 claims 1
- 230000001629 suppression Effects 0.000 description 2
- 230000035945 sensitivity Effects 0.000 description 1
Description
これらのおよび他の態様は、本開示を全体として考慮すると、当業者に容易に明らかとなろう。
特定の実施形態では、例えば以下が提供される:
(項目1)
ハンチントン病(HD)を有する被験体においてニューロンを改変する方法であって、該ニューロンが改変されるように、該被験体に変異体Htt(mHtt)対立遺伝子の抑制因子を投与することを含む、方法。
(項目2)
前記ニューロンは、HDニューロンである、項目1に記載の方法。
(項目3)
前記改変は、前記ニューロンにおけるHttの凝集を低減すること;該ニューロンにおけるエネルギー代謝を増加させること;該ニューロンにおけるアポトーシスに対する感受性を低減すること、およびそれらの組合せを含む、項目1または項目2に記載の方法。
(項目4)
細胞内のATPレベルが増加する、項目3に記載の方法。
(項目5)
HDニューロンにおけるHtt凝集体の形成を防止または低減する方法であって、項目3に記載の方法に従って該HDニューロンを改変することを含む、方法。
(項目6)
HDニューロンにおける細胞活性を増加させる方法であって、項目3に記載の方法に従って該HDニューロンを改変することを含む、方法。
(項目7)
HDニューロンにおけるアポトーシスを低減する方法であって、項目3に記載の方法に従って該HDニューロンを改変することを含む、方法。
(項目8)
運動障害を低減することを必要とするHD被験体において運動障害を低減するための方法であって、項目2に記載の方法に従って該被験体におけるHDニューロンを改変することを含む、方法。
(項目9)
前記運動障害は、クラスピングを含む、項目8に記載の方法。
(項目10)
前記被験体におけるmHttレベルを検出することをさらに含む、項目1から9のいずれかに記載の方法。
(項目11)
前記mHttレベルは、脳脊髄液(CSF)中で検出される、項目10に記載の方法。
(項目12)
前記抑制因子は、ジンクフィンガータンパク質転写因子(ZF)−TF)、TAL−エフェクタードメイン転写因子(TALE−TF)、CRISPR/Cas−転写因子、ジンクフィンガーヌクレアーゼ(ZFN)、TAL−エフェクタードメインヌクレアーゼ(TALEN)またはCRISPR/Casヌクレアーゼ系を含む、項目1から11のいずれかに記載の方法。
(項目13)
前記抑制因子は、タンパク質として、またはポリヌクレオチドとして前記ニューロンに送達される、項目1から12のいずれかに記載の方法。
(項目14)
前記ポリヌクレオチドは、mRNA、またはプラスミドおよびウイルスベクターからなる群より選択される発現構築物を含む、項目13に記載の方法。
(項目15)
前記抑制因子は、mHtt発現を少なくとも85%低減させる、項目1から14のいずれかに記載の方法。
(項目16)
Htt抑制因子、およびmHttタンパク質のレベルを検出および/または定量するための試薬を含むキット。
特定の実施形態では、例えば以下が提供される:
(項目1)
ハンチントン病(HD)を有する被験体においてニューロンを改変する方法であって、該ニューロンが改変されるように、該被験体に変異体Htt(mHtt)対立遺伝子の抑制因子を投与することを含む、方法。
(項目2)
前記ニューロンは、HDニューロンである、項目1に記載の方法。
(項目3)
前記改変は、前記ニューロンにおけるHttの凝集を低減すること;該ニューロンにおけるエネルギー代謝を増加させること;該ニューロンにおけるアポトーシスに対する感受性を低減すること、およびそれらの組合せを含む、項目1または項目2に記載の方法。
(項目4)
細胞内のATPレベルが増加する、項目3に記載の方法。
(項目5)
HDニューロンにおけるHtt凝集体の形成を防止または低減する方法であって、項目3に記載の方法に従って該HDニューロンを改変することを含む、方法。
(項目6)
HDニューロンにおける細胞活性を増加させる方法であって、項目3に記載の方法に従って該HDニューロンを改変することを含む、方法。
(項目7)
HDニューロンにおけるアポトーシスを低減する方法であって、項目3に記載の方法に従って該HDニューロンを改変することを含む、方法。
(項目8)
運動障害を低減することを必要とするHD被験体において運動障害を低減するための方法であって、項目2に記載の方法に従って該被験体におけるHDニューロンを改変することを含む、方法。
(項目9)
前記運動障害は、クラスピングを含む、項目8に記載の方法。
(項目10)
前記被験体におけるmHttレベルを検出することをさらに含む、項目1から9のいずれかに記載の方法。
(項目11)
前記mHttレベルは、脳脊髄液(CSF)中で検出される、項目10に記載の方法。
(項目12)
前記抑制因子は、ジンクフィンガータンパク質転写因子(ZF)−TF)、TAL−エフェクタードメイン転写因子(TALE−TF)、CRISPR/Cas−転写因子、ジンクフィンガーヌクレアーゼ(ZFN)、TAL−エフェクタードメインヌクレアーゼ(TALEN)またはCRISPR/Casヌクレアーゼ系を含む、項目1から11のいずれかに記載の方法。
(項目13)
前記抑制因子は、タンパク質として、またはポリヌクレオチドとして前記ニューロンに送達される、項目1から12のいずれかに記載の方法。
(項目14)
前記ポリヌクレオチドは、mRNA、またはプラスミドおよびウイルスベクターからなる群より選択される発現構築物を含む、項目13に記載の方法。
(項目15)
前記抑制因子は、mHtt発現を少なくとも85%低減させる、項目1から14のいずれかに記載の方法。
(項目16)
Htt抑制因子、およびmHttタンパク質のレベルを検出および/または定量するための試薬を含むキット。
Claims (16)
- ハンチントン病(HD)を有する被験体においてニューロンを改変するための組成物であって、変異体Htt(mHtt)対立遺伝子の抑制因子を含み、該改変は、該ニューロンにおけるHttの凝集を低減すること;該ニューロンにおけるエネルギー代謝を増加させること;該ニューロンにおけるアポトーシスに対する感受性を低減すること、またはそれらの組合せを含む、組成物。
- 前記ニューロンは、HDニューロンである、請求項1に記載の組成物。
- 前記改変は、前記ニューロンにおけるHttの凝集を低減することを含む、請求項1または請求項2に記載の組成物。
- 細胞内のATPレベルが増加する、請求項3に記載の組成物。
- HDニューロンにおけるHtt凝集体の形成を防止または低減するための請求項3に記載の組成物。
- HDニューロンにおける細胞活性を増加させるための請求項3に記載の組成物。
- HDニューロンにおけるアポトーシスを低減するための請求項3に記載の組成物。
- 運動障害を低減することを必要とするHD被験体において運動障害を低減するための請求項2に記載の組成物。
- 前記運動障害は、クラスピングを含む、請求項8に記載の組成物。
- 前記被験体におけるmHttレベルが検出されることを特徴とする、請求項1から9のいずれかに記載の組成物。
- 前記mHttレベルが脳脊髄液(CSF)中で検出されることを特徴とする、請求項10に記載の組成物。
- 前記抑制因子は、ジンクフィンガータンパク質転写因子(ZF)−TF)、TAL−エフェクタードメイン転写因子(TALE−TF)、CRISPR/Cas−転写因子、ジンクフィンガーヌクレアーゼ(ZFN)、TAL−エフェクタードメインヌクレアーゼ(TALEN)またはCRISPR/Casヌクレアーゼ系を含む、請求項1から11のいずれかに記載の組成物。
- 前記抑制因子がタンパク質として、またはポリヌクレオチドとして前記ニューロンに送達されることを特徴とする、請求項1から12のいずれかに記載の組成物。
- 前記ポリヌクレオチドは、mRNA、またはプラスミドおよびウイルスベクターからなる群より選択される発現構築物を含む、請求項13に記載の組成物。
- 前記抑制因子は、mHtt発現を少なくとも85%低減させる、請求項1から14のいずれかに記載の組成物。
- Htt抑制因子、およびmHttタンパク質のレベルを検出および/または定量するための試薬を含むキット。
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 (ja) | 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 (13)
Country | Link |
---|---|
US (1) | US11110154B2 (ja) |
EP (1) | EP3139966B1 (ja) |
JP (1) | JP2017515819A (ja) |
KR (1) | KR102380324B1 (ja) |
CN (1) | CN106413760B (ja) |
AU (1) | AU2015255877B2 (ja) |
BR (1) | BR112016025849A2 (ja) |
CA (1) | CA2947035A1 (ja) |
HK (1) | HK1232141A1 (ja) |
MX (1) | MX2016014565A (ja) |
NZ (1) | NZ725542A (ja) |
RU (1) | RU2691102C2 (ja) |
WO (1) | WO2015171932A1 (ja) |
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