MicroRNA and diseases: Therapeutic potential as new generation of drugs

Wahid, Fazli; Khan, Taous; Kim, You Young

doi:10.1016/j.biochi.2014.05.004

Cited by 51 publications

(31 citation statements)

References 223 publications

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“…MiR-based therapies have been emerging in the last few years. Indeed, some clinical trials using miR-based therapies are being performed [61]. Nevertheless, one of the major concerns in the translation of miR-based therapies to the clinic is the efficient delivery of these molecules into cells.…”

Section: Discussionmentioning

confidence: 99%

MicroRNA-124 loaded nanoparticles enhance brain repair in Parkinson's disease

Saraiva

Paiva

Santos

et al. 2016

Journal of Controlled Release

143

128

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Modulation of the subventricular zone (SVZ) neurogenic niche can enhance brain repair in several disorders including Parkinson's disease (PD). Herein, we used biocompatible and traceable polymeric nanoparticles (NPs) containing perfluoro-1,5-crown ether (PFCE) and coated with protamine sulfate to complex microRNA-124 (miR-124), a neuronal fate determinant. The ability of NPs to efficiently deliver miR-124 and prompt SVZ neurogenesis and brain repair in PD was evaluated. In vitro, miR-124 NPs were efficiently internalized by neural stem/progenitors cells and neuroblasts and promoted their neuronal commitment and maturation. The expression of Sox9 and Jagged1, two miR-124 targets and stemness-related genes, were also decreased upon miR-124 NP treatment. In vivo, the intracerebral administration of miR-124 NPs increased the number of migrating neuroblasts that reached the granule cell layer of the olfactory bulb, both in healthy and in a 6-hydroxydopamine (6-OHDA) mouse model for PD.MiR-124 NPs were also able to induce migration of neurons into the lesioned striatum of 6-OHDA-treated mice. Most importantly, miR-124 NPs proved to ameliorate motor symptoms of 6-OHDA mice, monitored by the apomorphine-induced rotation test.Altogether, we provide clear evidences to support the use of miR-124 NPs as a new therapeutic approach to boost endogenous brain repair mechanisms in a setting of neurodegeneration.

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Section: Discussionmentioning

confidence: 99%

MicroRNA-124 loaded nanoparticles enhance brain repair in Parkinson's disease

Saraiva

Paiva

Santos

et al. 2016

Journal of Controlled Release

143

128

View full text Add to dashboard Cite

show abstract

“…binding to the 3′-untranslated regions (3′-UTRs) of target messenger RNAs (mRNAs), resulting in the degradation of mRNAs or repression of their translation [6]. Beyond the involvement in diverse biological processes, accumulating evidence has demonstrated that miRNAs are frequently deregulated in various human malignancies, which are involved in cancer cell proliferation, apoptosis, angiogenesis, invasion, metastasis, etc.…”

Section: Introductionmentioning

confidence: 99%

miR-383 inhibits hepatocellular carcinoma cell proliferation via targeting APRIL

Chen¹,

Guan²,

Gu³

et al. 2015

Tumor Biol.

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Mounting evidence has shown that microRNAs (miRNAs), a class of small non-coding RNAs, are frequently deregulated in human malignancies and have pivotal roles in diverse biological processes including cancer cell proliferation. Herein, we investigated the expression pattern of miR-383 in 64 hepatocellular carcinoma (HCC) tissues and 4 HCC cell lines and found that miR-383 was downregulated in HCC tissues and cell lines. Moreover, miR-383 expression in HCC was significantly correlated with tumor size and tumor-node-metastasis (TNM) stage. Kaplan-Meier analysis showed that decreased miR-383 expression was associated with poor overall survival of HCC patients. In addition, Cox regression analysis indicated that miR-383 was an independent prognostic factor for HCC patients. Then, functional studies demonstrated that ectopic miR-383 expression could significantly suppress the in vitro proliferation of HCC cells, as well as induce cell cycle arrest and cell apoptosis. Luciferase reporter assay further identified that a proliferation-inducing ligand (APRIL), a member in the tumor necrosis factor (TNF) superfamily, was a novel target gene for miR-383. Subsequent investigation revealed that miR-383 expression was inversely correlated with APRIL messenger RNA (mRNA) expression in HCC tissues. Besides, recombinant human APRIL (rhAPRIL) could rescue HCC cell proliferation inhibited by miR-383. Taken together, our present study provided the first evidence that miR-383 was decreased in HCC and associated with tumor progression and prognosis of HCC patients. Furthermore, our findings confirmed that miR-383 might inhibit HCC cell proliferation partially via downregulating APRIL expression. Thus, this study might provide a promising strategy by targeting with the miR-383-APRIL axis in the treatment of HCC.

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“…Also, the US Food and Drug Administration recently approved a sensitive noninvasive screening assay for colorectal cancer that examines a panel of markers, including KRAS mutations and NDRG4 and BMP3 methylation status [126]. An array of additional epigenetic tests is either available or in development for a very broad range of diseases, including many nervous system disorders [127][128][129][130][131][132][133][134]. These assays are purported to have roles in risk stratification, screening, prognostication, customization of therapies, and monitoring treatment responses and disease recurrence; however, data from prospective trials is limited.…”

Section: Perspectivementioning

confidence: 99%

Epigenetic Mechanisms Underlying the Pathogenesis of Neurogenetic Diseases

Qureshi

Mehler

2014

Neurotherapeutics

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There have been considerable advances in uncovering the complex genetic mechanisms that underlie nervous system disease pathogenesis, particularly with the advent of exome and whole genome sequencing techniques. The emerging field of epigenetics is also providing further insights into these mechanisms. Here, we discuss our understanding of the interplay that exists between genetic and epigenetic mechanisms in these disorders, highlighting the nascent field of epigenetic epidemiology-which focuses on analyzing relationships between the epigenome and environmental exposures, development and aging, other healthrelated phenotypes, and disease states-and next-generation research tools (i.e., those leveraging synthetic and chemical biology and optogenetics) for examining precisely how epigenetic modifications at specific genomic sites affect disease processes.

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MicroRNA and diseases: Therapeutic potential as new generation of drugs

Cited by 51 publications

References 223 publications

MicroRNA-124 loaded nanoparticles enhance brain repair in Parkinson's disease

MicroRNA-124 loaded nanoparticles enhance brain repair in Parkinson's disease

miR-383 inhibits hepatocellular carcinoma cell proliferation via targeting APRIL

Epigenetic Mechanisms Underlying the Pathogenesis of Neurogenetic Diseases

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