通过降低基于锁核酸的剪接寡核苷酸的二级结构含量来增强外显子跳跃活性。
Enhancement of exon skipping activity by reduction in the secondary structure content of LNA-based splice-switching oligonucleotides.
机构信息
Graduate School of Pharmaceutical Sciences, Osaka University, 1-6, Yamadaoka, Suita, Osaka, 565-0871, Japan.
出版信息
Chem Commun (Camb). 2019 Jun 11;55(48):6850-6853. doi: 10.1039/c8cc09648a.
PMID:31123731
Abstract
PAGE and UV melting analysis revealed that longer LNA-based splice-switching oligonucleotides (SSOs) formed secondary structures by themselves, reducing their effective concentration. To avoid such secondary structure formation, we introduced 7-deaza-2'-deoxyguanosine or 2'-deoxyinosine into the SSOs. These modified SSOs, with fewer secondary structures, showed higher exon skipping activities.
摘要
PAGE 和 UV 融解分析显示，较长的基于锁核酸的剪接寡核苷酸（SSO）会自行形成二级结构，从而降低其有效浓度。为了避免这种二级结构的形成，我们在 SSO 中引入了 7-脱氮-2'-脱氧鸟苷或 2'-脱氧肌苷。这些修饰后的 SSO 具有较少的二级结构，显示出更高的外显子跳跃活性。
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