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 (enExample) | 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 (24)
| Country | Link |
|---|---|
| US (3) | US10435441B2 (enExample) |
| EP (1) | EP3352776B1 (enExample) |
| JP (2) | JP6853257B2 (enExample) |
| KR (1) | KR102803519B1 (enExample) |
| CN (1) | CN108348576B (enExample) |
| AU (2) | AU2016325613B2 (enExample) |
| BR (1) | BR112018005779A2 (enExample) |
| CA (1) | CA2998500A1 (enExample) |
| CL (1) | CL2018000749A1 (enExample) |
| DK (1) | DK3352776T3 (enExample) |
| EA (1) | EA037993B1 (enExample) |
| ES (1) | ES3032775T3 (enExample) |
| FI (1) | FI3352776T3 (enExample) |
| HR (1) | HRP20250728T1 (enExample) |
| HU (1) | HUE071655T2 (enExample) |
| IL (1) | IL258030B (enExample) |
| LT (1) | LT3352776T (enExample) |
| MX (1) | MX391052B (enExample) |
| PL (1) | PL3352776T3 (enExample) |
| PT (1) | PT3352776T (enExample) |
| RS (1) | RS66924B1 (enExample) |
| SI (1) | SI3352776T1 (enExample) |
| WO (1) | WO2017053753A1 (enExample) |
| ZA (2) | ZA201801679B (enExample) |
Families Citing this family (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| SI3352776T1 (sl) * | 2015-09-23 | 2025-08-29 | Sangamo Therapeutics, Inc. | Represorji Htt in njihove uporabe |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| CN109804066A (zh) | 2016-08-09 | 2019-05-24 | 哈佛大学的校长及成员们 | 可编程cas9-重组酶融合蛋白及其用途 |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | 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 |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| 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 |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| EP3716767A4 (en) * | 2017-10-24 | 2021-11-24 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| WO2019204457A1 (en) * | 2018-04-18 | 2019-10-24 | Sangamo Therapeutics, Inc. | Zinc finger protein compositions for modulation of huntingtin (htt) |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| CN113301909B (zh) * | 2019-01-15 | 2025-01-28 | 桑格摩生物治疗股份有限公司 | Htt阻遏物及其应用 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| JP2023525304A (ja) | 2020-05-08 | 2023-06-15 | ザ ブロード インスティテュート，インコーポレーテッド | 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物 |
| 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 SI SI201631908T patent/SI3352776T1/sl unknown
- 2016-09-23 JP JP2018534513A patent/JP6853257B2/ja active Active
- 2016-09-23 PT PT168497212T patent/PT3352776T/pt unknown
- 2016-09-23 FI FIEP16849721.2T patent/FI3352776T3/fi active
- 2016-09-23 PL PL16849721.2T patent/PL3352776T3/pl unknown
- 2016-09-23 WO PCT/US2016/053374 patent/WO2017053753A1/en not_active Ceased
- 2016-09-23 RS RS20250597A patent/RS66924B1/sr unknown
- 2016-09-23 KR KR1020187009994A patent/KR102803519B1/ko active Active
- 2016-09-23 IL IL258030A patent/IL258030B/en unknown
- 2016-09-23 AU AU2016325613A patent/AU2016325613B2/en active Active
- 2016-09-23 CA CA2998500A patent/CA2998500A1/en active Pending
- 2016-09-23 MX MX2018003491A patent/MX391052B/es unknown
- 2016-09-23 DK DK16849721.2T patent/DK3352776T3/da active
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