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1篇 您的检索式:作者名="Shuaiwei Ren"
    题名 作者 年代 出处 被引量
1Efficient Modulation of Exon Skipping via Antisense Circular RNAs显示文摘Splice-switching antisense oligonucleotides(ASOs)and engineered U7 small nuclear ribonucleoprotein(U7 Sm OPT)are the most commonly used methods for exon skipping.However,challenges remain,such as limited organ delivery and repeated dosing for ASOs and unknown risks of by-products produced by U7 Sm OPT.Here,we showed that antisense circular RNAs(AS-circRNAs)can effectively mediate exon skipping in both minigene and endogenous transcripts.We also showed a relatively higher exon skipping efficiency at the tested Dmd minigene than U7 Sm OPT.AS-circRNA specifically targets the precursor mRNA splicing without off-target effects.Moreover,AS-circRNAs with adeno-associated virus(AAV)delivery corrected the open reading frame and restored the dystrophin expression in a mouse model of Duchenne muscular dystrophy.In conclusion,we develop an alternative method for regulating RNA splicing,which might be served as a novel tool for genetic disease treatment.Shuaiwei Ren Mei Huang Raoxian Bai Lijiao Chen Jiao Yang Junyu Zhang Wenting Guo Weizhi Ji Yongchang Chen 2023Research2023,,3:0
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