2015
DOI: 10.1055/s-0034-1397352
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Adeno-Associated Virus-Mediated MicroRNA Delivery and Therapeutics

Abstract: MicroRNAs are 20-24 nt long, single-stranded RNAs that repress gene expression. Dysregulation of miRNA expression is associated with many human diseases. Modulating the level of endogenous miRNA alters gene profiling and can achieve therapeutic benefits. Here, we reviewed currently used methods of altering miRNA activity in vivo. We focus on the delivery of miRNAs and miRNA inhibitors using recombinant adeno-associated virus (rAAV). In general, rAAV-mediated miRNA inhibition or overexpression provides a simple… Show more

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Cited by 27 publications
(19 citation statements)
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“…Baculovirus-mediated miR delivery offers unique advantages because baculovirus cannot proliferate in mammalian hosts and because baculovirus degrades in the host over time, making this methodology a promising therapeutic approach [ 84 ]. Presently, AAV is the most encouraging viral system for gene therapy, as it can prolong transgene expression in its host and can transduce dividing and non-dividing cells [ 84 , 87 ]. AAV is non-pathogenic and thus poses a minimal threat to immune systems in humans.…”
Section: Strategies For Therapeutic Deliverymentioning
confidence: 99%
“…Baculovirus-mediated miR delivery offers unique advantages because baculovirus cannot proliferate in mammalian hosts and because baculovirus degrades in the host over time, making this methodology a promising therapeutic approach [ 84 ]. Presently, AAV is the most encouraging viral system for gene therapy, as it can prolong transgene expression in its host and can transduce dividing and non-dividing cells [ 84 , 87 ]. AAV is non-pathogenic and thus poses a minimal threat to immune systems in humans.…”
Section: Strategies For Therapeutic Deliverymentioning
confidence: 99%
“…Although the adenovirus-mediated delivery of TNA seems straightforward, there are a number of fundamental limitations such as the size of the construct for expression and the existence of an adaptive immune response by B cells or CD8 T cells in a significant number of people. 154 Therefore, downregulation of ncRNA in the liver is preferable by the use of chemically modified TNA and alternative nonviral delivery systems. The liver is the main target organ for systemic delivery of modified TNA due to efficient mechanisms of receptor-mediated delivery.…”
Section: Targeting Mirna and Lncrna In The Livermentioning
confidence: 99%
“…Viral vectors have been utilised in order to deliver miR mimics or inhibitors. For example, adeno-associated virus (AAV) is a popular gene delivery system; however, a consistent issue which has inhibited the progression of some AAV therapies to human clinical trials is the induction of low grade immune activation (118). Lenti-viral vectors for delivery suffer from the same immunogenicity issues.…”
Section: Microrna Therapeuticsmentioning
confidence: 99%