CN109963945A - 单一向导rna、crispr/cas9系统及其使用方法 - Google Patents

单一向导rna、crispr/cas9系统及其使用方法 Download PDF

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Publication number
CN109963945A
CN109963945A CN201780064151.5A CN201780064151A CN109963945A CN 109963945 A CN109963945 A CN 109963945A CN 201780064151 A CN201780064151 A CN 201780064151A CN 109963945 A CN109963945 A CN 109963945A
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sequence
mutation
dna
artificial sequence
snp
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Chinese (zh)
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T.摩尔
A.内斯比特
D.考特尼
K.克里斯蒂
G.李
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Avellino Lab USA Inc
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Avellino Lab USA Inc
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CN201780064151.5A 2016-08-20 2017-08-21 单一向导rna、crispr/cas9系统及其使用方法 Pending CN109963945A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662377586P 2016-08-20 2016-08-20
US62/377,586 2016-08-20
US201762462808P 2017-02-23 2017-02-23
US62/462,808 2017-02-23
US201762501750P 2017-05-05 2017-05-05
US62/501,750 2017-05-05
PCT/US2017/047861 WO2018039145A1 (en) 2016-08-20 2017-08-21 Single guide rna, crispr/cas9 systems, and methods of use thereof

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CN109963945A true CN109963945A (zh) 2019-07-02

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US (1) US20190185850A1 (https=)
EP (1) EP3500677A4 (https=)
JP (3) JP2019524149A (https=)
KR (2) KR102594051B1 (https=)
CN (1) CN109963945A (https=)
WO (1) WO2018039145A1 (https=)

Cited By (4)

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CN110499335A (zh) * 2019-08-08 2019-11-26 复旦大学 CRISPR/SauriCas9基因编辑系统及其应用
CN110551763A (zh) * 2019-08-08 2019-12-10 复旦大学 CRISPR/SlutCas9基因编辑系统及其应用
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
CN119464347A (zh) * 2024-11-26 2025-02-18 扬州大学 基于CRISPR-Cas9系统的噬菌体基因组编辑载体及其编辑方法和应用

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US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
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EP4036228A1 (en) 2015-11-13 2022-08-03 Avellino Lab USA, Inc. Methods for the treatment of corneal dystrophies
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
JP2019524149A (ja) * 2016-08-20 2019-09-05 アベリノ ラボ ユーエスエー インコーポレイテッドＡｖｅｌｌｉｎｏ Ｌａｂ ＵＳＡ， Ｉｎｃ． 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
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US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US12390514B2 (en) 2017-03-09 2025-08-19 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
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KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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
WO2018225807A1 (ja) * 2017-06-07 2018-12-13 国立大学法人東京大学 顆粒状角膜変性症に対する遺伝子治療薬
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
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
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
US20210032612A1 (en) * 2018-02-22 2021-02-04 Avellino Lab Usa, Inc. CRISPR/Cas9 Systems, and Methods of Use Thereof
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