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Updated: Jun 18, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
07:02

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

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专为纠正异常拼接而设计的定制的反意义寡核酸揭示了罕见遗传疾病可操作的突变组

Htoo A Wai1, Eliska Svobodova1,2,3, Natalia Romero Herrera1

  • 1Human Development and Health, Faculty of Medicine, University of Southampton, Southampton, UK.

Experimental & molecular medicine
|July 31, 2024
PubMed
概括

反感性寡核酸为治疗罕见遗传疾病提供了快速有效的方法. 这项研究成功地纠正了五种不同的异常拼接突变,为个性化治疗铺平了道路.

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Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

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相关概念视频

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Alternative RNA Splicing

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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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