Oligonucleotide Analogues as Modulators of the Expression and Function of Noncoding RNAs (ncRNAs): Emerging Therapeutics Applications

被引：10

作者：

Avitabile, Concetta ^{[1
]}

Cimmino, Amelia ^{[2
,3
]}

Romanelli, Alessandra ^{[1
]}

机构：

[1] Univ Naples Federico II, Dipartimento Farm, I-80134 Naples, Italy

[2] CNR, Ist Genet & Biofis Adriano Buzzati Traverso, I-80131 Naples, Italy

[3] Ist Ricovero & Cura Carattere Sci SDN IRCC, I-80142 Naples, Italy

来源：

JOURNAL OF MEDICINAL CHEMISTRY | 2014年 / 57卷 / 24期

关键词：

NUCLEIC-ACIDS PNA; ANTI-MIRNA OLIGONUCLEOTIDES; IN-VIVO; MESSENGER-RNAS; ANTISENSE OLIGONUCLEOTIDES; CHROMOSOMAL DELETIONS; NONHUMAN-PRIMATES; MICRORNA ACTIVITY; CRYSTAL-STRUCTURE; GENE;

D O I：

10.1021/jm5006594

中图分类号：

R914 [药物化学];

学科分类号：

100701 ;

摘要：

ncRNAs are emerging as key regulators of physiological and pathological processes and therefore have been identified as pharmacological targets and as markers for some diseases. Oligonucleotide analogues represent so far the most widely employed tool for the modulation of the expression of ncRNAs. In this perspective we briefly describe most of the known classes of ncRNAs and then we discuss the design and the applications of oligonucleotide analogues for their targeting. The effects of modifications of the chemical structure of the oligonucleotides on properties such as the binding affinity toward targets and off targets, and the stability to degradation and their biological effects (when known) are discussed. Examples of molecules currently used in clinical trials are also reported.

引用

收藏

页码：10220 / 10240

页数：21

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