Research (Wash D C). 2023 Jan 19
Shuaiwei Ren, Mei Huang, Raoxian Bai, Lijiao Chen, Jiao Yang, Junyu Zhang, Wenting Guo, Weizhi Ji, and Yongchang Chen
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Research Field: Exon skipping

AAV Serotype: MyoAAV

Dose: Two-week-old Dmd male mice were injected intraperitoneally with 100 μl of the virus at a dose of 5 × 1013 vg/kg. Control mice (2-week-old C57BL/6J male mice and Dmd male mice) were injected with an equal volume of saline. At least 3 mice were injected for each experimental condition. Mice were monitored 4 times per week for 4 weeks.

Routes of Administration: injected intraperitoneally

Targeted organ: muscle

Animal or cell line strain: mouse model of Duchenne muscular dystrophy.


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.

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