AU2016287836A1 - CRISPR/Cas9-based treatments - Google Patents
CRISPR/Cas9-based treatments Download PDFInfo
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- AU2016287836A1 AU2016287836A1 AU2016287836A AU2016287836A AU2016287836A1 AU 2016287836 A1 AU2016287836 A1 AU 2016287836A1 AU 2016287836 A AU2016287836 A AU 2016287836A AU 2016287836 A AU2016287836 A AU 2016287836A AU 2016287836 A1 AU2016287836 A1 AU 2016287836A1
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- dystrophy
- corneal dystrophy
- corneal
- pct
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12N15/90—Stable introduction of foreign DNA into chromosome
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
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| US201562188013P | 2015-07-02 | 2015-07-02 | |
| US62/188,013 | 2015-07-02 | ||
| PCT/US2016/040962 WO2017004616A1 (en) | 2015-07-02 | 2016-07-05 | Crispr/cas9-based treatments |
Publications (1)
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| AU2016287836A1 true AU2016287836A1 (en) | 2018-02-15 |
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| AU2016287836A Abandoned AU2016287836A1 (en) | 2015-07-02 | 2016-07-05 | CRISPR/Cas9-based treatments |
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| US (1) | US20200010854A1 (https=) |
| EP (1) | EP3317409A4 (https=) |
| JP (1) | JP2018520149A (https=) |
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| IL (1) | IL256279A (https=) |
| MX (1) | MX2017016921A (https=) |
| WO (1) | WO2017004616A1 (https=) |
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| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| DK3155101T3 (da) | 2014-06-16 | 2020-05-04 | Univ Johns Hopkins | Sammensætninger og fremgangsmåder til ekspression af CRISPR-leder-RNA´er under anvendelse af H1-promotoren |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| 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 |
| ES2905558T3 (es) * | 2015-11-13 | 2022-04-11 | Avellino Lab Usa Inc | Procedimientos para el tratamiento de las distrofias corneales |
| EP3445375A1 (en) * | 2016-04-22 | 2019-02-27 | Intellia Therapeutics, Inc. | Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four |
| 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 |
| WO2020046861A1 (en) * | 2018-08-27 | 2020-03-05 | Avellino Lab Usa, Inc. | Crispr/cas9 systems, and methods of use 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 |
| WO2018165541A1 (en) * | 2017-03-10 | 2018-09-13 | The Board Of Regents Of The University Of Texas System | Treatment of fuchs' endothelial corneal dystrophy |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| 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 |
| CN111065736A (zh) * | 2017-06-07 | 2020-04-24 | 国立大学法人东京大学 | 针对颗粒状角膜变性症的基因治疗药物 |
| 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 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| DE102017131324A1 (de) | 2017-12-27 | 2019-06-27 | Beckhoff Automation Gmbh | Statormodul und Planarantriebssystem |
| CN108949823B (zh) * | 2017-12-29 | 2020-02-14 | 广州医科大学附属第三医院（广州重症孕产妇救治中心、广州柔济医院） | 靶向敲除ATXN3基因中扩展突变型polyQ序列的方法 |
| US12480140B2 (en) | 2018-05-06 | 2025-11-25 | Emendobio Inc. | Differential knockout of an allele of a heterozygous ELANE gene |
| 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 |
| KR102126573B1 (ko) | 2018-10-18 | 2020-06-26 | 대한민국 | CRISPR-Cas9 시스템을 이용한 유색고약버섯의 리그닌 분해효소의 유전자 편집방법 및 이의 용도 |
| JP7216877B2 (ja) * | 2018-10-29 | 2023-02-02 | 中国▲農▼▲業▼大学 | 新規なＣＲＩＳＰＲ／Ｃａｓ１２ｆ酵素およびシステム |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| 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 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| JP2022551970A (ja) * | 2019-10-16 | 2022-12-14 | ブラウン ユニバーシティ | 筋再生および筋成長 |
| 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 |
| CN111849991B (zh) * | 2020-08-05 | 2022-04-08 | 武汉纽福斯生物科技有限公司 | 一种寡核苷酸及其应用 |
| EP4430185A1 (en) * | 2021-11-09 | 2024-09-18 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy |
| WO2023092132A1 (en) * | 2021-11-22 | 2023-05-25 | Mammoth Biosciences, Inc. | Effector proteins and uses thereof |
| WO2024097900A1 (en) * | 2022-11-02 | 2024-05-10 | Emendobio Inc. | Compositions and methods for transcription factor 4 (tcf4) repeat expansion excision |
| CN116286823A (zh) * | 2023-04-04 | 2023-06-23 | 尧唐(上海)生物科技有限公司 | 靶向TGFBI基因的sgRNA、载体及其应用 |
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| DK2931898T3 (en) * | 2012-12-12 | 2016-06-20 | Massachusetts Inst Technology | CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| AU2014361781B2 (en) * | 2013-12-12 | 2021-04-01 | Massachusetts Institute Of Technology | Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing |
| MX2016007325A (es) * | 2013-12-12 | 2017-07-19 | Broad Inst Inc | Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. |
| EP3445375A1 (en) * | 2016-04-22 | 2019-02-27 | Intellia Therapeutics, Inc. | Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four |
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2016
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- 2016-07-05 BR BR112017028201A patent/BR112017028201A2/pt not_active Application Discontinuation
- 2016-07-05 WO PCT/US2016/040962 patent/WO2017004616A1/en not_active Ceased
- 2016-07-05 MX MX2017016921A patent/MX2017016921A/es unknown
- 2016-07-05 EA EA201890203A patent/EA201890203A1/ru unknown
- 2016-07-05 EP EP16818953.8A patent/EP3317409A4/en not_active Withdrawn
- 2016-07-05 AU AU2016287836A patent/AU2016287836A1/en not_active Abandoned
- 2016-07-05 JP JP2017567629A patent/JP2018520149A/ja active Pending
- 2016-07-05 CN CN201680050474.4A patent/CN108350446A/zh active Pending
- 2016-07-05 US US15/741,444 patent/US20200010854A1/en not_active Abandoned
- 2016-07-05 KR KR1020187002971A patent/KR20180041120A/ko not_active Withdrawn
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2017
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- 2017-12-27 CL CL2017003411A patent/CL2017003411A1/es unknown
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| MX2017016921A (es) | 2018-04-10 |
| EP3317409A1 (en) | 2018-05-09 |
| CA2989331A1 (en) | 2017-01-05 |
| KR20180041120A (ko) | 2018-04-23 |
| CN108350446A (zh) | 2018-07-31 |
| CL2017003411A1 (es) | 2018-08-17 |
| BR112017028201A2 (pt) | 2018-08-28 |
| IL256279A (en) | 2018-02-28 |
| US20200010854A1 (en) | 2020-01-09 |
| EP3317409A4 (en) | 2019-02-20 |
| WO2017004616A1 (en) | 2017-01-05 |
| JP2018520149A (ja) | 2018-07-26 |
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