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首页> 外文会议>Oligonucleotide Therapeutics Society >Characterization of Antisense Oligonucleotides Comprising 2'-Deoxy-2'-Fluoro-β-D-Arabinonucleic Acid (FANA) Specificity, Potency, and Duration of Activity
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Characterization of Antisense Oligonucleotides Comprising 2'-Deoxy-2'-Fluoro-β-D-Arabinonucleic Acid (FANA) Specificity, Potency, and Duration of Activity

机译：反义寡核苷酸的表征包含2'-脱氧-2'-氟-β-D-亚丙核酸（FANA）特异性，效力和活性持续时间的

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Antisense oligonucleotides (AON) are being developed for a wide array of therapeutic applications. Significant improvements in their serum stability, target affinity, and safety profile have been achieved with the development of chemically modified oligonucleotides. Here, we com-pared 2'-deoxy-2'-fluoro-?-D-arabinonucleic acid (FANA)-containing AONs with phosphorothioate oligodeoxynucleotides (PS-DNA), V-O- methyl-RNA/DNA chimeras and short interfering RNAs (siRNA) with respect to their target knockdown efficacy, duration of action and re-sistance to nuclease degradation. Results show that two different con-figurations of FANA/DNA chimeras (altimers and gapmers) were found to have potent antisense activity. Specific target inhibition was observed with both FANA configurations with an estimated EC_(50) value comparable to that of an siRNA but 20-to 100-fold lower than the other commonly used AONs. Moreover, the FANA/DNA chimeras showed increased serum stability that was correlated with sustained antisense activity for up to 4 days. Taken together, these results indicate that chimeric FANA/DNA AONs are promising new tools for therapeutic gene silencing when in-creased potency and duration of action are required.
机译：反义寡核苷酸（AON）正被用于广泛的治疗应用开发的。在他们的血清稳定性，靶亲和力，和安全性的改善显著已经获得了化学修饰的寡核苷酸的发展。在这里，我们COM-削减2'-脱氧-2'-氟 - - d-arabinonucleic酸（FANA）含具有硫代磷酸酯寡脱氧核苷酸（PS-DNA），VO-甲基-RNA / DNA嵌合体和短干扰RNA的AON（ siRNA）的相对于它们的目标敲低的功效，作用和再sistance的持续时间对核酸酶降解。结果表明，发现FANA / DNA嵌合体（altimers和间隔体）的两个不同的CON-音型具有有效的反义活性。用比得上siRNA的但20至100倍比其它常用的AON下既FANA配置与估计EC_（50）值观察到特异性靶抑制。此外，FANA / DNA嵌合体显示增加的，将其用为长达4天的持续的反义活性相关的血清稳定性。总之，这些结果表明，嵌合FANA / DNA的AON是有前途的新工具，用于治疗的基因沉默时折痕效力和作用持续时间是必需的。

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