Covalent Strategies for Targeting Messenger and Non-Coding RNAs: An Updated Review on siRNA, miRNA and antimiR Conjugates.
Covalent Strategies for Targeting Messenger and Non-Coding RNAs: An Updated Review on siRNA, miRNA and antimiR Conjugates.
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DOI：
10.3390/genes9020074
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发表时间：
2018-02-06
期刊：
影响因子：
3.5
通讯作者：
Eritja R
中科院分区：
文献类型：
--
作者：
Grijalvo S;Alagia A;Jorge AF;Eritja R
在寻找涉及基因相关疾病的新疗法时，基于寡核苷酸的疗法已成为经典方法的替代方案。已经描述了几种机制，其中证明了寡核苷酸对于调节基因表达的关键作用。反义寡核苷酸 (ASO) 以及最近的 siRNA 和 miRNA 在通过序列特异性靶向信使 RNA (mRNA) 降低异常蛋白水平或恢复与包括癌症在内的许多疾病有关的非编码 RNA (ncRNA) 的异常水平方面做出了重要贡献。除了有助于加速 ASO、siRNA 和 miRNA 在临床试验中的存在的配方方法之外；非病毒载体和核酸之间的共价连接也增加了价值，并为开发有前途的基于核酸的疗法开辟了新的前景。这篇综述文章主要关注共价修饰 siRNA 和 miRNA 分子的策略。讨论了用于合成 siRNA 缀合物的细胞穿透肽 (CPP)、碳水化合物、聚合物、脂质和适体的例子，而在基于 miRNA 的药物的情况下，这篇综述文章特别强调使用 antagomir、锁核酸 (LNA)、肽核酸 (PNA) 以及纳米颗粒。还讨论了 siRNA 和 miRNA 缀合物的生物医学应用。
Oligonucleotide-based therapy has become an alternative to classical approaches in the search of novel therapeutics involving gene-related diseases. Several mechanisms have been described in which demonstrate the pivotal role of oligonucleotide for modulating gene expression. Antisense oligonucleotides (ASOs) and more recently siRNAs and miRNAs have made important contributions either in reducing aberrant protein levels by sequence-specific targeting messenger RNAs (mRNAs) or restoring the anomalous levels of non-coding RNAs (ncRNAs) that are involved in a good number of diseases including cancer. In addition to formulation approaches which have contributed to accelerate the presence of ASOs, siRNAs and miRNAs in clinical trials; the covalent linkage between non-viral vectors and nucleic acids has also added value and opened new perspectives to the development of promising nucleic acid-based therapeutics. This review article is mainly focused on the strategies carried out for covalently modifying siRNA and miRNA molecules. Examples involving cell-penetrating peptides (CPPs), carbohydrates, polymers, lipids and aptamers are discussed for the synthesis of siRNA conjugates whereas in the case of miRNA-based drugs, this review article makes special emphasis in using antagomiRs, locked nucleic acids (LNAs), peptide nucleic acids (PNAs) as well as nanoparticles. The biomedical applications of siRNA and miRNA conjugates are also discussed.
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影响因子：
46.9
作者：
Alvarez-Erviti, Lydia;Seow, Yiqi;Wood, Matthew J. A.
通讯作者：
Wood, Matthew J. A.
DOI：
10.1016/j.omtn.2017.06.005
发表时间：
2017-09-15
期刊：
Molecular therapy. Nucleic acids
影响因子：
--
作者：
Chakraborty C;Sharma AR;Sharma G;Doss CGP;Lee SS
通讯作者：
Lee SS
影响因子：
20.1
作者：
Corsten, Maarten F.;Papageorgiou, Anna;Heymans, Stephane
通讯作者：
Heymans, Stephane
谷胱甘肽响应纳米载体作为靶向细胞内药物和基因递送的有前途的平台
影响因子：
10.8
作者：
Cheng, Ru;Feng, Fang;Zhong, Zhiyuan
通讯作者：
Zhong, Zhiyuan
影响因子：
4.3
作者：
Campuzano, Susana;Pedrero, Maria;Pingarron, Jose M.
通讯作者：
Pingarron, Jose M.