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.)
- Granted
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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 (cg-RX-API-DMAC7.html) | 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 (23)
Families Citing this family (40)
| Publication number | Priority date | Publication date | Assignee | Title |
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| CA2853829C (en) | 2011-07-22 | 2023-09-26 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | 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 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| 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 |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| EP3177718B1 (en) | 2014-07-30 | 2022-03-16 | President and Fellows of Harvard College | Cas9 proteins including ligand-dependent inteins |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| RS66924B1 (sr) * | 2015-09-23 | 2025-07-31 | Sangamo Therapeutics Inc | Htt represori i njihova upotreba |
| SG11201803173VA (en) | 2015-10-23 | 2018-05-30 | Harvard College | Nucleobase editors and uses thereof |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| IL302748B1 (en) | 2016-05-18 | 2025-09-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | 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 | アデノシン核酸塩基編集因子およびそれらの使用 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| 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 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US11752181B2 (en) | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| AU2018352592C1 (en) | 2017-10-16 | 2025-09-25 | Beam Therapeutics, Inc. | Uses of adenosine base editors |
| AU2018355343B2 (en) * | 2017-10-24 | 2024-12-05 | 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 |
| US11421007B2 (en) | 2018-04-18 | 2022-08-23 | Sangamo Therapeutics, Inc. | Zinc finger protein compositions for modulation of huntingtin (Htt) |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | 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 |
| WO2020150338A1 (en) * | 2019-01-15 | 2020-07-23 | Sangamo Therapeutics, Inc. | Htt repressors and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst Inc | Methods and compositions for editing nucleotide sequences |
| 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 |
| 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 RS RS20250597A patent/RS66924B1/sr unknown
- 2016-09-23 ES ES16849721T patent/ES3032775T3/es active Active
- 2016-09-23 WO PCT/US2016/053374 patent/WO2017053753A1/en not_active Ceased
- 2016-09-23 PL PL16849721.2T patent/PL3352776T3/pl unknown
- 2016-09-23 SI SI201631908T patent/SI3352776T1/sl unknown
- 2016-09-23 IL IL258030A patent/IL258030B/en unknown
- 2016-09-23 DK DK16849721.2T patent/DK3352776T3/da active
- 2016-09-23 US US15/274,357 patent/US10435441B2/en active Active
- 2016-09-23 MX MX2018003491A patent/MX391052B/es unknown
- 2016-09-23 AU AU2016325613A patent/AU2016325613B2/en active Active
- 2016-09-23 JP JP2018534513A patent/JP6853257B2/ja active Active
- 2016-09-23 CN CN201680061809.2A patent/CN108348576B/zh active Active
- 2016-09-23 HU HUE16849721A patent/HUE071655T2/hu unknown
- 2016-09-23 KR KR1020187009994A patent/KR102803519B1/ko active Active
- 2016-09-23 HR HRP20250728TT patent/HRP20250728T1/hr unknown
- 2016-09-23 PT PT168497212T patent/PT3352776T/pt unknown
- 2016-09-23 EA EA201890493A patent/EA037993B1/ru unknown
- 2016-09-23 LT LTEPPCT/US2016/053374T patent/LT3352776T/lt unknown
- 2016-09-23 BR BR112018005779-7A patent/BR112018005779A2/pt active Search and Examination
- 2016-09-23 EP EP16849721.2A patent/EP3352776B1/en active Active
- 2016-09-23 FI FIEP16849721.2T patent/FI3352776T3/fi active
-
2018
- 2018-03-12 ZA ZA201801679A patent/ZA201801679B/en unknown
- 2018-03-21 CL CL2018000749A patent/CL2018000749A1/es unknown
-
2019
- 2019-05-27 ZA ZA2019/03325A patent/ZA201903325B/en unknown
- 2019-08-26 US US16/550,949 patent/US11123443B2/en active Active
-
2020
- 2020-06-17 AU AU2020204035A patent/AU2020204035B2/en active Active
- 2020-10-14 JP JP2020173022A patent/JP2021007405A/ja active Pending
-
2021
- 2021-08-13 US US17/402,233 patent/US20210369872A1/en active Pending
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