基于核酸nanotherapeutics穿越血脑屏障

Title:Nucleic Acids-based Nanotherapeutics Crossing the Blood Brain Barrier

Volume: 17 Issue: 2

关键词: 纳米载体，脑内传递，神经退行性疾病、脑胶质瘤、脑蛋白、基因传递、mRNA

摘要: The restless endeavors revealing the molecular pathways underlying many neurodegenerative diseases and brain tumors have paved the way for the introduction of the selective exogenous gene-based therapeutics. The implicated active biomolecules encompass mainly negatively-charged nucleic acids ranging from DNA, mRNA, non-coding RNAs (small-interfering RNA, siRNA, and microRNA, miRNA), to antisense oligonucleotides. They selectively interfere with the genes translational and/or transcriptional processes.

Although many reviews previously addressed brain targeting, a thorough correlation between the molecular properties of these biomacromolecules, the nature of blood brain barrier (BBB) in the accompanying pathological condition, the intracellular targets, as well as the design of the delivery system which will transport the bioactive cargo to the target cells attempting efficient delivery to the active sites in the brain will be appraised.

In this review, we will further discuss the tremendous advances in non-viral gene delivery nanosystems currently investigated (starting from self-assembled nanoplexes using cationic polymers or lipids and going through liposomes, aptamers, polymersomes, exosomes, dendrimers and nanoparticles). Unlike previous reviews on this topic, functionalization strategies of the nanocarriers promoting either surface receptor binding or intracellular targeting of the cranial cells will be highlighted, with special emphasis on tailoring smart nanomedicines according to the CNS disease condition. In addition, newly-developed evaluation approaches, cell culture models studying BBB permeability and manipulation of the barrier function of the brain via focused ultrasound will be addressed.

Cite this article as:

Nucleic Acids-based Nanotherapeutics Crossing the Blood Brain Barrier, Current Gene Therapy 2017; 17 (2) . https://dx.doi.org/10.2174/1566523217666170510155803

DOI https://dx.doi.org/10.2174/1566523217666170510155803	Print ISSN 1566-5232
Publisher Name Bentham Science Publisher	Online ISSN 1875-5631