期刊
CURRENT GENE THERAPY
卷 17, 期 2, 页码 105-119出版社
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1566523217666170510160527
关键词
Dendrimers; RNAi; siRNA; Gene therapy; In vivo; Nanomedicine; Nanoparticles; Biodistribution
资金
- Fundo para a Investigacao em Saude (INFARMED) [FIS-2015-01_CCV_20150630-88]
- FEDER funds through the Programa Operacional Competitividade e Internacionalizacao - COMPETE
- Portuguese funds through FCT - Fundacao para a Ciencia e a Tecnologia [POCI-01-0145-FEDER-016639, PTDC/CTM-NAN/3547/2014]
- Norte Portugal Regional Operational Programme through the European Regional Development Fund (ERDF) [NORTE-01-0145-FEDER-000012]
- FCT [SFRH/BPD/109297/2015]
- Fundação para a Ciência e a Tecnologia [SFRH/BPD/109297/2015, PTDC/CTM-NAN/3547/2014] Funding Source: FCT
Over the last decades, gene therapy has emerged as a pioneering therapeutic approach to treat or prevent several diseases. Among the explored strategies, the short-term silencing of protein coding genes mediated by siRNAs has a good therapeutic potential in a clinical setting. However, the widespread use of siRNA will require the development of clinically suitable, safe and effective vehicles with the ability to complex and deliver siRNA into target cells with minimal toxicity. Lately, dendrimers have gained considerable attention as non-viral vectors in nucleic acid delivery due to their unique structural characteristics (globular, well defined and highly branched structure, multivalency, low polydispersity and tunable nanosize), along with their relevant capacity to complex and protect nucleic acids in compact nanostructures, which can be functionalized with targeting moieties in order to get cell specificity. Here, we present an overview of the state-of-the-art of the most significant and recent advances on the use of dendrimers as siRNA delivery vectors, with particular focus on the in vivo applications. We will cover the use of different dendrimers, distinct administration routes, toxicity issues, as well as the target tissue or disease, highlighting the potential of dendrimers as nanocarriers for therapeutic and biomedical applications.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据