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Advancing ASO therapies from development to implementation
Nature Medicine ( IF 58.7 ) Pub Date : 2024-09-13 , DOI: 10.1038/s41591-024-03217-x Rebecca Schuele 1, 2 , Matthis Synofzik 2, 3 , Holm Graessner 4 , Annemieke Aartsma-Rus 5
Nature Medicine ( IF 58.7 ) Pub Date : 2024-09-13 , DOI: 10.1038/s41591-024-03217-x Rebecca Schuele 1, 2 , Matthis Synofzik 2, 3 , Holm Graessner 4 , Annemieke Aartsma-Rus 5
Affiliation
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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.
中文翻译：
推进 ASO 疗法从开发到实施
为治疗单个患者而开发的反义寡核苷酸 （ASO） 技术的新应用增加了罕见病的“个性化”疗法的数量;但迫切需要实施和获取的途径。
更新日期：2024-09-13
中文翻译：
推进 ASO 疗法从开发到实施
为治疗单个患者而开发的反义寡核苷酸 （ASO） 技术的新应用增加了罕见病的“个性化”疗法的数量;但迫切需要实施和获取的途径。
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