Advancing ASO therapies from development to implementation

IF 58.7 1区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Nature Medicine Pub Date : 2024-09-13 DOI:10.1038/s41591-024-03217-x

Rebecca Schuele, Matthis Synofzik, Holm Graessner, Annemieke Aartsma-Rus

引用次数: 0

Abstract

A novel application of antisense oligonucleotide (ASO) technology, developed to treat a single patient, adds to the growing number of ‘personalized’ therapies for rare diseases; but pathways to implementation and access are urgently needed.

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推进 ASO 疗法从开发到实施的进程

反义寡核苷酸 (ASO) 技术的一种新应用是为治疗单个患者而开发的，它为越来越多的罕见病 "个性化 "疗法增添了新的内容；但实施和获取途径亟待开发。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Nature Medicine 医学-生化与分子生物学

CiteScore

100.90

自引率

0.70%

发文量

525

审稿时长

1 months

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