Massachusetts General Hospital Cancer Center, Charlestown, MA 02129, USA.
Department of Cell Biology, Harvard Medical School, Boston, MA 02115, USA.
Sci Transl Med. 2013 Nov 20;5(212):212ra162. doi: 10.1126/scitranslmed.3006840.
MicroRNAs (miRNAs) regulate many aspects of human biology. They target mRNAs for translational repression or degradation through base pairing with 3' untranslated regions, primarily via seed sequences (nucleotides 2 to 8 in the mature miRNA sequence). A number of individual miRNAs and miRNA families share seed sequences and targets, but differ in the sequences outside of the seed. miRNAs have been implicated in the etiology of a wide variety of human diseases and therefore represent promising therapeutic targets. However, potential redundancy of different miRNAs sharing the same seed sequence and the challenge of simultaneously targeting miRNAs that differ significantly in nonseed sequences complicate therapeutic targeting approaches. We recently demonstrated effective inhibition of entire miRNA families using seed-targeting 8-mer locked nucleic acid (LNA)-modified antimiRs in short-term experiments in mammalian cells and in mice. However, the long-term efficacy and safety of this approach in higher organisms, such as humans and nonhuman primates, have not been determined. We show that pharmacological inhibition of the miR-33 family, key regulators of cholesterol/lipid homeostasis, by a subcutaneously delivered 8-mer LNA-modified antimiR in obese and insulin-resistant nonhuman primates results in derepression of miR-33 targets, such as ABCA1, increases circulating high-density lipoprotein cholesterol, and is well tolerated over 108 days of treatment. These findings demonstrate the efficacy and safety of an 8-mer LNA-antimiR against an miRNA family in a nonhuman primate metabolic disease model, suggesting that this could be a feasible approach for therapeutic targeting of miRNA families sharing the same seed sequence in human diseases.
微小 RNA(miRNAs)调节人类生物学的许多方面。它们通过与 3'非翻译区的碱基配对,主要通过种子序列(成熟 miRNA 序列中的第 2 到 8 个核苷酸)来靶向 mRNAs 进行翻译抑制或降解。许多单独的 miRNAs 和 miRNA 家族共享种子序列和靶标,但在种子序列之外的序列上有所不同。miRNAs 与多种人类疾病的病因有关,因此代表了有前途的治疗靶点。然而,不同 miRNA 之间存在潜在的冗余性,它们共享相同的种子序列,并且同时靶向非种子序列差异很大的 miRNA 具有挑战性,这使得治疗靶向方法变得复杂。我们最近在哺乳动物细胞和小鼠的短期实验中证明,使用种子靶向 8 聚体锁核酸(LNA)修饰的抗 miRNA 可以有效抑制整个 miRNA 家族。然而,这种方法在高等生物(如人类和非人类灵长类动物)中的长期疗效和安全性尚未确定。我们表明,通过皮下给药的 8 聚体 LNA 修饰的抗 miRNA 抑制 miR-33 家族(胆固醇/脂质稳态的关键调节剂),可导致 miR-33 靶标如 ABCA1 的去抑制,增加循环高密度脂蛋白胆固醇,并在 108 天的治疗中耐受良好。这些发现证明了在非人类灵长类动物代谢疾病模型中使用 8 聚体 LNA-抗 miRNA 针对 miRNA 家族的有效性和安全性,表明这可能是针对人类疾病中具有相同种子序列的 miRNA 家族进行治疗靶向的可行方法。