JP2018532428A5 - - Google Patents
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- JP2018532428A5 JP2018532428A5 JP2018534513A JP2018534513A JP2018532428A5 JP 2018532428 A5 JP2018532428 A5 JP 2018532428A5 JP 2018534513 A JP2018534513 A JP 2018534513A JP 2018534513 A JP2018534513 A JP 2018534513A JP 2018532428 A5 JP2018532428 A5 JP 2018532428A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- zinc finger
- domain
- item
- cell
- 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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- 102000040430 polynucleotide Human genes 0.000 claims description 12
- 108091033319 polynucleotide Proteins 0.000 claims description 12
- 239000002157 polynucleotide Substances 0.000 claims description 12
- 239000013607 AAV vector Substances 0.000 claims description 10
- 101710185494 Zinc finger protein Proteins 0.000 claims description 10
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 10
- 101150043003 Htt gene Proteins 0.000 claims description 8
- 210000004027 cell Anatomy 0.000 claims description 8
- 108020001507 fusion proteins Proteins 0.000 claims description 8
- 102000037865 fusion proteins Human genes 0.000 claims description 8
- 239000008194 pharmaceutical composition Substances 0.000 claims description 7
- 210000002569 neuron Anatomy 0.000 claims description 6
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims description 4
- 210000004556 brain Anatomy 0.000 claims description 4
- 238000013518 transcription Methods 0.000 claims description 4
- 230000035897 transcription Effects 0.000 claims description 4
- 239000011701 zinc Substances 0.000 claims description 4
- 229910052725 zinc Inorganic materials 0.000 claims description 4
- 108700028369 Alleles Proteins 0.000 claims description 2
- 208000023105 Huntington disease Diseases 0.000 claims description 2
- 101710163270 Nuclease Proteins 0.000 claims description 2
- 230000004913 activation Effects 0.000 claims description 2
- 210000001577 neostriatum Anatomy 0.000 claims description 2
- 230000002265 prevention Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 11
- 238000000034 method Methods 0.000 description 9
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562222588P | 2015-09-23 | 2015-09-23 | |
| US62/222,588 | 2015-09-23 | ||
| PCT/US2016/053374 WO2017053753A1 (en) | 2015-09-23 | 2016-09-23 | Htt repressors and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020173022A Division JP2021007405A (ja) | 2015-09-23 | 2020-10-14 | Ｈｔｔリプレッサー及びその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018532428A JP2018532428A (ja) | 2018-11-08 |
| JP2018532428A5 true JP2018532428A5 (https=) | 2019-09-26 |
| JP6853257B2 JP6853257B2 (ja) | 2021-03-31 |
Family
ID=58387548
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018534513A Active JP6853257B2 (ja) | 2015-09-23 | 2016-09-23 | Ｈｔｔリプレッサー及びその使用 |
| JP2020173022A Pending JP2021007405A (ja) | 2015-09-23 | 2020-10-14 | Ｈｔｔリプレッサー及びその使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020173022A Pending JP2021007405A (ja) | 2015-09-23 | 2020-10-14 | Ｈｔｔリプレッサー及びその使用 |
Country Status (22)
| Country | Link |
|---|---|
| US (3) | US10435441B2 (https=) |
| EP (1) | EP3352776B1 (https=) |
| JP (2) | JP6853257B2 (https=) |
| KR (1) | KR102803519B1 (https=) |
| CN (1) | CN108348576B (https=) |
| AU (2) | AU2016325613B2 (https=) |
| CL (1) | CL2018000749A1 (https=) |
| DK (1) | DK3352776T3 (https=) |
| EA (1) | EA037993B1 (https=) |
| ES (1) | ES3032775T3 (https=) |
| FI (1) | FI3352776T3 (https=) |
| HR (1) | HRP20250728T1 (https=) |
| HU (1) | HUE071655T2 (https=) |
| IL (1) | IL258030B (https=) |
| LT (1) | LT3352776T (https=) |
| MX (1) | MX391052B (https=) |
| PL (1) | PL3352776T3 (https=) |
| PT (1) | PT3352776T (https=) |
| RS (1) | RS66924B1 (https=) |
| SI (1) | SI3352776T1 (https=) |
| WO (1) | WO2017053753A1 (https=) |
| ZA (2) | ZA201801679B (https=) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| CN119876138A (zh) | 2014-11-14 | 2025-04-25 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2017053753A1 (en) * | 2015-09-23 | 2017-03-30 | Sangamo Biosciences, Inc. | Htt repressors and uses thereof |
| JP7109784B2 (ja) | 2015-10-23 | 2022-08-01 | 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 | 遺伝子編集のための進化したＣａｓ９蛋白質 |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| CA3024449A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| KR102547316B1 (ko) | 2016-08-03 | 2023-06-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| EP3526320A1 (en) | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| JP2020510038A (ja) | 2017-03-09 | 2020-04-02 | 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 | がんワクチン |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| CA3079727A1 (en) * | 2017-10-24 | 2019-05-02 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of rare diseases |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| WO2019204457A1 (en) * | 2018-04-18 | 2019-10-24 | Sangamo Therapeutics, Inc. | Zinc finger protein compositions for modulation of huntingtin (htt) |
| EP3797160A1 (en) | 2018-05-23 | 2021-03-31 | The Broad Institute Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| EP3911349A4 (en) * | 2019-01-15 | 2023-01-18 | Sangamo Therapeutics, Inc. | HTT REPRESSORS AND THEIR USES |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| JP7624728B2 (ja) | 2019-02-25 | 2025-01-31 | ユニバーシティ オブ マサチューセッツ | Ｄｎａ結合ドメイントランスアクチベーター及びその使用 |
| JP7657726B2 (ja) | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート，インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| EP4004206A1 (en) * | 2019-07-23 | 2022-06-01 | University of Rochester | Targeted rna cleavage with crispr-cas |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
| WO2025004001A1 (en) * | 2023-06-30 | 2025-01-02 | Takeda Pharmaceutical Company Limited | Htt repressors and uses thereof |
| KR102868476B1 (ko) | 2025-02-20 | 2025-10-13 | 본아이에프 주식회사 | 냄비용 모듈형 교반기 |
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-
2016
- 2016-09-23 WO PCT/US2016/053374 patent/WO2017053753A1/en not_active Ceased
- 2016-09-23 ES ES16849721T patent/ES3032775T3/es active Active
- 2016-09-23 EP EP16849721.2A patent/EP3352776B1/en active Active
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