首页> 外国专利> METHODS FOR PERFORMING ANTISENSE OLIGONUCLEOTIDE-MEDIATED EXON SKIPPING IN THE RETINA OF A SUBJECT IN NEED THEREOF

METHODS FOR PERFORMING ANTISENSE OLIGONUCLEOTIDE-MEDIATED EXON SKIPPING IN THE RETINA OF A SUBJECT IN NEED THEREOF

机译：需要在受试者的视网膜中进行反义寡核苷酸介导的外显子跳跃的方法

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摘要

The present invention relates to methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof. In particular, the present invention relates to a method for performing antisense oligonucleotide-mediated exon skipping in a retina cell of a subject comprising the step of injecting into the vitreous of the subject an amount of the antisense oligonucleotide.

机译：本发明涉及在有需要的受试者的视网膜中进行反义寡核苷酸介导的外显子跳跃的方法。特别地，本发明涉及一种在受试者的视网膜细胞中进行反义寡核苷酸介导的外显子跳跃的方法，其包括将一定量的反义寡核苷酸注射到受试者的玻璃体内的步骤。

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公开/公告号EP3702461A1

专利类型
公开/公告日2020-09-02

原文格式PDF
申请/专利权人 INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE - INSERM;UNIVERSITÉ DE PARIS;FONDATION IMAGINE;ASSISTANCE PUBLIQUE HOPITAUX DE PARIS;
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申请/专利号EP20200168215
发明设计人 ROZET JEAN-MICHEL;PERRAULT ISABELLE;GERARD XAVIER;KAPLAN JOSSELINE;MUNNICH ARNOLD;
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申请日2014-07-08
分类号C12N15/113;A61K31/7088;
国家 EP
入库时间 2022-08-21 11:39:16

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