推进 ASO 疗法从开发到实施的进程

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

摘要

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。

Advancing ASO therapies from development to implementation

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Advancing ASO therapies from development to implementation

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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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

期刊介绍： Nature Medicine is a monthly journal publishing original peer-reviewed research in all areas of medicine. The publication focuses on originality, timeliness, interdisciplinary interest, and the impact on improving human health. In addition to research articles, Nature Medicine also publishes commissioned content such as News, Reviews, and Perspectives. This content aims to provide context for the latest advances in translational and clinical research, reaching a wide audience of M.D. and Ph.D. readers. All editorial decisions for the journal are made by a team of full-time professional editors. Nature Medicine consider all types of clinical research, including: -Case-reports and small case series -Clinical trials, whether phase 1, 2, 3 or 4 -Observational studies -Meta-analyses -Biomarker studies -Public and global health studies Nature Medicine is also committed to facilitating communication between translational and clinical researchers. As such, we consider “hybrid” studies with preclinical and translational findings reported alongside data from clinical studies.