ES2673028T3 - Reducción selectiva de la actividad perjudicial de genes que contienen repeticiones de trinucleótidos prolongadas - Google Patents

Reducción selectiva de la actividad perjudicial de genes que contienen repeticiones de trinucleótidos prolongadas Download PDF

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ES2673028T3
ES2673028T3 ES11847833T ES11847833T ES2673028T3 ES 2673028 T3 ES2673028 T3 ES 2673028T3 ES 11847833 T ES11847833 T ES 11847833T ES 11847833 T ES11847833 T ES 11847833T ES 2673028 T3 ES2673028 T3 ES 2673028T3
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genes
trinucleotide repeats
selective reduction
prolonged
harmful activity
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Tzu-Hao Cheng
Chia-Rung Liu
Tzu-Han Wang
Stanley N. Cohen
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National Yang Ming Chiao Tung University NYCU
Leland Stanford Junior University
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National Yang Ming Chiao Tung University NYCU
Leland Stanford Junior University
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Abstract

Un agente para el uso en la modificación de la progresión en un sujeto de una enfermedad que surge de un gen que contiene un dominio de repeticiones de trinucleótidos prolongadas mutantes, en el que la administración al sujeto de una cantidad eficaz del agente reduce de manera selectiva una actividad transcripcional mediada por SPT4, para reducir de manera selectiva la actividad perjudicial del gen para modificar la progresión de la enfermedad en el sujeto.
ES11847833T 2010-12-10 2011-12-08 Reducción selectiva de la actividad perjudicial de genes que contienen repeticiones de trinucleótidos prolongadas Active ES2673028T3 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
TW99143336 2010-12-10
US13/110,494 US8569254B2 (en) 2010-12-10 2011-05-18 Methods for modulating the expression and aggregation of CAG-expanded gene product in cells and methods for identifying agents useful for doing the same
US201161513970P 2011-08-01 2011-08-01
PCT/US2011/063997 WO2012078906A2 (en) 2010-12-10 2011-12-08 Selective reduction of the deleterious activity of extended tri-nucleotide repeat containing genes

Publications (1)

Publication Number Publication Date
ES2673028T3 true ES2673028T3 (es) 2018-06-19

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ES11847833T Active ES2673028T3 (es) 2010-12-10 2011-12-08 Reducción selectiva de la actividad perjudicial de genes que contienen repeticiones de trinucleótidos prolongadas

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US (6) US8569254B2 (es)
EP (2) EP2463372B1 (es)
JP (1) JP5769084B2 (es)
ES (1) ES2673028T3 (es)
TR (1) TR201808029T4 (es)
TW (1) TWI430812B (es)
WO (1) WO2012078906A2 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
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US8569254B2 (en) 2010-12-10 2013-10-29 National Yang Ming University Methods for modulating the expression and aggregation of CAG-expanded gene product in cells and methods for identifying agents useful for doing the same
AU2016270297B2 (en) 2015-05-29 2019-12-12 National Yang-Ming University Nucleoside agents for the reduction of the deleterious activity of extended nucleotide repeat containing genes
JP7089763B2 (ja) * 2015-10-05 2022-06-23 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ トリヌクレオチドリピート伸長が関与する遺伝的疾患の予防または治療における一本鎖アンチセンスオリゴヌクレオチドの使用
AU2018286393A1 (en) * 2017-06-15 2020-01-30 College Of Medicine Pochon Cha University Industry-Academic Cooperation Foundation Genome editing system for repeat expansion mutation
EP3641758A4 (en) * 2017-06-19 2021-03-17 The Board of Trustees of the Leland Stanford Junior University Compounds for the reduction of the deleterious activity of extended nucleotide repeat containing genes
WO2019141279A1 (en) * 2018-01-22 2019-07-25 Taiwan Strategics Intellectual Property Co. Ltd. Method to enhance the transcription regulation of SUPT4H on genes containing repetitive nucleotide sequences
US20210238226A1 (en) * 2018-05-22 2021-08-05 Design Therapeutics, Inc. Methods and compounds for the treatment of genetic disease

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Also Published As

Publication number Publication date
JP5769084B2 (ja) 2015-08-26
WO2012078906A3 (en) 2012-10-04
US9226935B2 (en) 2016-01-05
US9637741B2 (en) 2017-05-02
JP2012125240A (ja) 2012-07-05
EP2463372B1 (en) 2015-09-02
US20130317088A1 (en) 2013-11-28
TR201808029T4 (tr) 2018-06-21
US10760077B2 (en) 2020-09-01
WO2012078906A2 (en) 2012-06-14
US20120149754A1 (en) 2012-06-14
TWI430812B (zh) 2014-03-21
US8569254B2 (en) 2013-10-29
TW201223545A (en) 2012-06-16
US9862947B2 (en) 2018-01-09
EP2705151A4 (en) 2014-10-08
EP2463372A1 (en) 2012-06-13
US9211303B2 (en) 2015-12-15
EP2705151B1 (en) 2018-03-21
US20160152978A1 (en) 2016-06-02
US20130331437A1 (en) 2013-12-12
US20180171336A1 (en) 2018-06-21
EP2705151A2 (en) 2014-03-12
US20160116456A1 (en) 2016-04-28

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