Nanoparticle-mediated delivery of therapeutic genes: focus on miRNA therapeutics
Source: Expert Opinion on Drug Delivery, Volume 10, Number 9, September 2013 , pp. 1259-1273(15)
Publisher: Informa Healthcare
Abstract:Introduction: Micro RNAs (miRNA) are 21 – 23 nucleotides long and regulate the expression of coding genes by binding imperfectly with their 3′ UTR region. The miRNA profile is altered in pathological processes, making miRNAs good targets for drug therapy. Restoration of down-regulated miRNA or inhibition of overexpressed miRNA to return miRNA to its normal state is the basis of miRNA-based therapy. This review focuses on nanocarriers used for the delivery of miRNA that confer physical stability to the unstable RNA structure, protect the RNA from nuclease degradation and aid in effective silencing of target genes.
Areas covered: The necessity of the nanocarrier for the delivery of the miRNA is emphasized and the recent research on liposome-, metal- and polymer-mediated miRNA delivery for the inhibition or replacement of the disease-related miRNA is summarized.
Expert opinion: The size, charge and surface properties of nanocarriers have to be tuned to ensure effective and safe delivery of the miRNA in clinical practice. The immune responses related to the nanocarriers and the double-stranded nucleotide delivery remain to be addressed. Also, the binding of miRNAs to non-specific targets has to be studied in more detail because miRNAs have multiple targets due to partial binding unlike siRNA.
Document Type: Research Article
Affiliations: 1: 1 Chonnam National University Medical School, Research Institute of Medical Sciences, Department of Biomedical Science, Gwangju 501-746, South Korea +82 61 379 8481; +82 61 379 8455;, Email: email@example.com 2: 3 Seoul National University, Department of Agricultural Biotechnology and Research Institute for Agriculture and Life Sciences, Seoul 151-921, South Korea +82 2 880 4868; +82 2 875 2494;, Email: firstname.lastname@example.org
Publication date: September 1, 2013