Orphan drugs, including antisense oligonucleotides (AONs), siRNAs/miRNAs, Cas9 nuclease, and recombinant genes, have recently been made available for rare diseases. However, the main bottleneck for these new therapies is delivery. Drugs/synthetic genes need to reach the affected tissues with minimal off-target effects and immune reactions. AON molecules are currently delivered as backboned naked compounds or via viral vectors. Nanocarriers are considered promising vehicles, able to improve drug distribution by organ targeting and limiting safety issues.

中文翻译：

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壳聚糖壳纳米气泡不可逆地包裹吗啉共轭反义寡核苷酸，对磷二酰胺吗啉介导的 DUX4 基因沉默无效

孤儿药，包括反义寡核苷酸 (AON)、siRNA/miRNA、Cas9 核酸酶和重组基因，最近已用于治疗罕见疾病。然而，这些新疗法的主要瓶颈是交付。药物/合成基因需要以最小的脱靶效应和免疫反应到达受影响的组织。AON 分子目前作为骨架裸化合物或通过病毒载体递送。纳米载体被认为是有前途的载体，能够通过器官靶向和限制安全问题来改善药物分布。