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MODULATEURS SPÉCIFIQUES ALLÉLIQUES DE LA RHODOPSINE P23H
专利权人:
Inc.;Ionis Pharmaceuticals
发明人:
申请号:
EP16756422.8
公开号:
EP3261643A1
申请日:
2016.02.26
申请国别(地区):
EP
年份:
2018
代理人:
摘要:
The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject. The present embodiments provided herein are directed to compounds and compositions useful for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP). In certain embodiments, P23H rhodopsin inhibitors provided herein are allele-specific antisense compounds targeted to a P23H mutant allele that are capable of selectively inhibiting expression of P23H rhodopsin mutant protein to a greater extent than wild-type protein. In certain embodiments, administration of the allele specific antisense compounds in a subject having AdRP results in selective inhibition of P23H rhodopsin and allows the normal protein produced from the wild-type allele to maintain rod survival and function in the subject.Les modes de réalisation de la présente invention concernent des procédés, des composés et des compositions permettant de traiter, prévenir, améliorer ou ralentir la progression de la rétinite pigmentaire (RP), telle que la rétinite pigmentaire autosomique dominante (AdRP) par l'administration d'un inhibiteur spécifique de la rhodopsine P23H à un sujet.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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