KR20180134412A - 전사 인자 4에서 트라이뉴클레오타이드 반복부와 연관된 질환의 치료를 위한 조성물 및 방법 - Google Patents

전사 인자 4에서 트라이뉴클레오타이드 반복부와 연관된 질환의 치료를 위한 조성물 및 방법 Download PDF

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KR20180134412A
KR20180134412A KR1020187033525A KR20187033525A KR20180134412A KR 20180134412 A KR20180134412 A KR 20180134412A KR 1020187033525 A KR1020187033525 A KR 1020187033525A KR 20187033525 A KR20187033525 A KR 20187033525A KR 20180134412 A KR20180134412 A KR 20180134412A
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seq
nuclease
guide rna
sequence
grna
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Korean (ko)
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션 마이클 번스
브래들리 앤드류 머레이
사라 베쓰 헤세
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인텔리아 테라퓨틱스, 인크.
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N15/09—Recombinant DNA-technology
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    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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  • Gastroenterology & Hepatology (AREA)
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KR1020187033525A 2016-04-22 2017-04-21 전사 인자 4에서 트라이뉴클레오타이드 반복부와 연관된 질환의 치료를 위한 조성물 및 방법 Ceased KR20180134412A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662326700P 2016-04-22 2016-04-22
US62/326,700 2016-04-22
PCT/US2017/028981 WO2017185054A1 (en) 2016-04-22 2017-04-21 Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four

Publications (1)

Publication Number Publication Date
KR20180134412A true KR20180134412A (ko) 2018-12-18

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KR1020187033525A Ceased KR20180134412A (ko) 2016-04-22 2017-04-21 전사 인자 4에서 트라이뉴클레오타이드 반복부와 연관된 질환의 치료를 위한 조성물 및 방법

Country Status (11)

Country Link
US (2) US20190142972A1 (https=)
EP (1) EP3445375A1 (https=)
JP (1) JP2019515914A (https=)
KR (1) KR20180134412A (https=)
CN (1) CN109414450A (https=)
AU (1) AU2017254718A1 (https=)
BR (1) BR112018071439A2 (https=)
CA (1) CA3021647A1 (https=)
CO (1) CO2018012433A2 (https=)
MX (1) MX2018012873A (https=)
WO (1) WO2017185054A1 (https=)

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CA2989331A1 (en) * 2015-07-02 2017-01-05 The Johns Hopkins University Crispr/cas9-based treatments
WO2022072458A1 (en) * 2020-09-29 2022-04-07 Avellino Lab Usa, Inc. Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy
AU2017379073B2 (en) 2016-12-22 2023-12-14 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2018165541A1 (en) * 2017-03-10 2018-09-13 The Board Of Regents Of The University Of Texas System Treatment of fuchs' endothelial corneal dystrophy
EP3723813A4 (en) * 2017-12-15 2021-10-06 Regents of the University of Minnesota GENOMIC CRISPR MEDIATION EDITING WITH VECTORS
CN109929872A (zh) * 2017-12-18 2019-06-25 中国科学院遗传与发育生物学研究所 一种通过基因编辑技术创制番茄白果材料的方法
WO2019122302A1 (en) 2017-12-21 2019-06-27 Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft Nucleic acid sequence replacement by nhej
US20190151470A1 (en) * 2018-01-21 2019-05-23 RoverMed BioSciences, LLC Nanoparticles comprising protein-polynucleotide complexes and for delivering protein based complexes
KR20210139225A (ko) * 2019-02-28 2021-11-22 프리드리히 이. 크루제 세포외 기질 조정제
AU2020229871B2 (en) * 2019-02-28 2023-05-18 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
JP2023515710A (ja) * 2020-04-27 2023-04-13 デューク ユニバーシティ ＣＲＩＳＰＲ媒介式エクソン欠失用の最適なｇＲＮＡ対を発見するためのハイスループットスクリーニング法
CN111944813B (zh) * 2020-08-24 2022-07-15 武汉纽福斯生物科技有限公司 核苷酸、病毒载体及其应用和RNAi药物制剂
CN111944814B (zh) * 2020-08-24 2022-07-15 武汉纽福斯生物科技有限公司 寡核苷酸、病毒载体及其应用和RNAi药物制剂
CR20230305A (es) 2020-12-11 2023-11-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación
EP4288541A1 (en) 2021-02-05 2023-12-13 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
EP4430185A1 (en) * 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
US12297285B2 (en) 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
CN120390656A (zh) 2022-11-08 2025-07-29 欧纳医疗公司 环状rna组合物
WO2024226536A1 (en) * 2023-04-24 2024-10-31 The General Hospital Corporation Methods and compositions for modifying genetic repeats
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

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WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
WO2013101711A1 (en) * 2011-12-30 2013-07-04 Mayo Foundation For Medical Education And Research Assessing likelihood of developing fuchs' corneal dystrophy
EP3241902B1 (en) * 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP2931899A1 (en) * 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP3608308B1 (en) 2013-03-08 2021-07-21 Novartis AG Lipids and lipid compositions for the delivery of active agents
AU2014346559B2 (en) * 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
EP3470089A1 (en) * 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
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CA2989331A1 (en) * 2015-07-02 2017-01-05 The Johns Hopkins University Crispr/cas9-based treatments

Also Published As

Publication number Publication date
US20250025580A1 (en) 2025-01-23
JP2019515914A (ja) 2019-06-13
CN109414450A (zh) 2019-03-01
BR112018071439A2 (pt) 2019-03-19
AU2017254718A1 (en) 2018-11-15
CO2018012433A2 (es) 2019-04-30
MX2018012873A (es) 2019-08-05
EP3445375A1 (en) 2019-02-27
CA3021647A1 (en) 2017-10-26
WO2017185054A1 (en) 2017-10-26
US20190142972A1 (en) 2019-05-16

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