Antisense Oligonucleotides: A Unique Treatment Approach

被引：10

作者：

Krishnan, Abhinaya V. ^{[1
]}

Mishra, Devendra ^{[1
]}

机构：

[1] Univ Delhi, Maulana Azad Med Coll, Dept Pediat, Delhi 110002, India

来源：

INDIAN PEDIATRICS | 2020年 / 57卷 / 02期

关键词：

Duchenne muscular dystrophy; Eteplirsen; Nusinersen; Spinal muscular atrophy; Treatment; SHAM CONTROL; NUSINERSEN; THERAPIES; SAFETY; TRIAL;

D O I：

10.1007/s13312-020-1736-7

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

Synthetic Antisense oligonucleotides (ASOs) are novel and efficient laboratory tools to regulate the expression of specific genes, and have only recently come into clinical use. These are synthetic single-stranded DNA analogs, whose sequence is complementary to a target nucleotide and alter protein synthesis by several mechanisms. We herein provide a primer on the topic for pediatricians, as this group of drugs is likely to see many more drugs for previously incurable diseases.

引用

页码：165 / 171

页数：7

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