CN108350446A - 基于crispr/cas9的治疗 - Google Patents

基于crispr/cas9的治疗 Download PDF

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Publication number
CN108350446A
CN108350446A CN201680050474.4A CN201680050474A CN108350446A CN 108350446 A CN108350446 A CN 108350446A CN 201680050474 A CN201680050474 A CN 201680050474A CN 108350446 A CN108350446 A CN 108350446A
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dystrophy
corneal dystrophy
syndrome
cas9
crispr
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CN201680050474.4A
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Chinese (zh)
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A.S.军
V.贾斯库拉-兰加
D.扎克
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Johns Hopkins University
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Johns Hopkins University
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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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    • 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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CN201680050474.4A 2015-07-02 2016-07-05 基于crispr/cas9的治疗 Pending CN108350446A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562188013P 2015-07-02 2015-07-02
US62/188013 2015-07-02
PCT/US2016/040962 WO2017004616A1 (en) 2015-07-02 2016-07-05 Crispr/cas9-based treatments

Publications (1)

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CN108350446A true CN108350446A (zh) 2018-07-31

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US (1) US20200010854A1 (enExample)
EP (1) EP3317409A4 (enExample)
JP (1) JP2018520149A (enExample)
KR (1) KR20180041120A (enExample)
CN (1) CN108350446A (enExample)
AU (1) AU2016287836A1 (enExample)
BR (1) BR112017028201A2 (enExample)
CA (1) CA2989331A1 (enExample)
CL (1) CL2017003411A1 (enExample)
EA (1) EA201890203A1 (enExample)
IL (1) IL256279A (enExample)
MX (1) MX2017016921A (enExample)
WO (1) WO2017004616A1 (enExample)

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CN111757889A (zh) * 2018-10-29 2020-10-09 中国农业大学 新型CRISPR/Cas12f酶和系统
CN111849991A (zh) * 2020-08-05 2020-10-30 武汉纽福斯生物科技有限公司 一种寡核苷酸及其应用
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

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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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
CN106852157B (zh) 2014-06-16 2022-04-12 约翰斯·霍普金斯大学 用于使用h1启动子表达crispr向导rna的组合物和方法
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
EP3374502B1 (en) * 2015-11-13 2021-10-27 Avellino Lab USA, Inc. Methods for the treatment of corneal dystrophies
CN109414450A (zh) * 2016-04-22 2019-03-01 因特利亚治疗公司 用于治疗与转录因子4中三核苷酸重复相关的疾病的组合物和方法
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
EP3497214B1 (en) 2016-08-09 2023-06-28 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
WO2018071868A1 (en) 2016-10-14 2018-04-19 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
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
US11512312B2 (en) 2017-03-10 2022-11-29 The Board Of Regents Of The University Of Texas System Treatment of Fuchs' endothelial corneal dystrophy
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
JP7161730B2 (ja) * 2017-06-07 2022-10-27 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
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
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US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
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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
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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111757889A (zh) * 2018-10-29 2020-10-09 中国农业大学 新型CRISPR/Cas12f酶和系统
CN111849991A (zh) * 2020-08-05 2020-10-30 武汉纽福斯生物科技有限公司 一种寡核苷酸及其应用
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
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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EP3317409A4 (en) 2019-02-20
EP3317409A1 (en) 2018-05-09
KR20180041120A (ko) 2018-04-23
CA2989331A1 (en) 2017-01-05
EA201890203A1 (ru) 2018-07-31
IL256279A (en) 2018-02-28
AU2016287836A1 (en) 2018-02-15
US20200010854A1 (en) 2020-01-09
JP2018520149A (ja) 2018-07-26
WO2017004616A1 (en) 2017-01-05
MX2017016921A (es) 2018-04-10
CL2017003411A1 (es) 2018-08-17
BR112017028201A2 (pt) 2018-08-28

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