CO2018012433A2 - Composiciones y métodos para tratar enfermedades asociadas con repeticiones de trinucleótidos en el factor de transcripción cuatro - Google Patents

Composiciones y métodos para tratar enfermedades asociadas con repeticiones de trinucleótidos en el factor de transcripción cuatro

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Publication number
CO2018012433A2
CO2018012433A2 CONC2018/0012433A CO2018012433A CO2018012433A2 CO 2018012433 A2 CO2018012433 A2 CO 2018012433A2 CO 2018012433 A CO2018012433 A CO 2018012433A CO 2018012433 A2 CO2018012433 A2 CO 2018012433A2
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Colombia
Prior art keywords
compositions
methods
trinucleotide repeats
transcription factor
diseases associated
Prior art date
Application number
CONC2018/0012433A
Other languages
English (en)
Inventor
Sean Michael Burns
Bradley Andrew Murray
Sarah Beth Hesse
Original Assignee
Intellia Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of CO2018012433A2 publication Critical patent/CO2018012433A2/es

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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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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 [CRISPRs]

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Abstract

La presente solicitud se refiere a composiciones y métodos para escindir repeticiones de trinucléotidos (TNR) contenidas en el intrón 3 de TCF4, tal como se observa en sujetos que tienen distrofia endotelial corneal de Fuchs (FECD), PSC y esquizofrenia. También se describen las composiciones que comprenden secuencias guías que se dirigen a la subunidad alfa 2 de colágeno VIII para el tratamiento de mutaciones allí que pueden contribuir a FECD.
CONC2018/0012433A 2016-04-22 2018-11-19 Composiciones y métodos para tratar enfermedades asociadas con repeticiones de trinucleótidos en el factor de transcripción cuatro CO2018012433A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662326700P 2016-04-22 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
CO2018012433A2 true CO2018012433A2 (es) 2019-04-30

Family

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Family Applications (1)

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CONC2018/0012433A CO2018012433A2 (es) 2016-04-22 2018-11-19 Composiciones y métodos para tratar enfermedades asociadas con repeticiones de trinucleótidos en el factor de transcripción cuatro

Country Status (11)

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

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KR20180041120A (ko) * 2015-07-02 2018-04-23 더 존스 홉킨스 유니버시티 Cprispr/cas9-기반 치료
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
WO2018165541A1 (en) * 2017-03-10 2018-09-13 The Board Of Regents Of The University Of Texas System Treatment of fuchs' endothelial corneal dystrophy
WO2019118875A1 (en) * 2017-12-15 2019-06-20 Ou Li Crispr-mediated genome 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
EP3728598A1 (en) 2017-12-21 2020-10-28 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
WO2020176747A1 (en) 2019-02-28 2020-09-03 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
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
AU2021394998A1 (en) 2020-12-11 2023-06-29 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination
WO2023086842A1 (en) * 2021-11-09 2023-05-19 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy

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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
PE20150336A1 (es) * 2012-05-25 2015-03-25 Univ California Metodos y composiciones para la modificacion de adn objetivo dirigida por arn y para la modulacion de la transcripcion dirigida por arn
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
KR102255108B1 (ko) 2013-03-08 2021-05-24 노파르티스 아게 활성제의 전달을 위한 지질 및 지질 조성물
CN106459995B (zh) * 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
KR20160089526A (ko) * 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 입자 전달 성분을 이용한 표적 장애 및 질병에 대한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 이용 및 치료 적용
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
EP3132030B1 (en) * 2014-04-18 2020-08-26 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
KR20180041120A (ko) * 2015-07-02 2018-04-23 더 존스 홉킨스 유니버시티 Cprispr/cas9-기반 치료

Also Published As

Publication number Publication date
AU2017254718A1 (en) 2018-11-15
CN109414450A (zh) 2019-03-01
KR20180134412A (ko) 2018-12-18
MX2018012873A (es) 2019-08-05
JP2019515914A (ja) 2019-06-13
EP3445375A1 (en) 2019-02-27
US20190142972A1 (en) 2019-05-16
WO2017185054A1 (en) 2017-10-26
CA3021647A1 (en) 2017-10-26
BR112018071439A2 (pt) 2019-03-19

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