Overexpression of microRNA-203 Suppresses Proliferation, Invasion, and Migration while Accelerating Apoptosis of CSCC Cell Line SCL-1.

abstract：:The acute hepatic porphyrias are caused by inherited enzymatic deficiencies in the heme biosynthesis pathway. Induction of the first enzyme 5-aminolevulinic acid synthase 1 (ALAS1) by triggers such as fasting or drug exposure can lead to accumulation of neurotoxic heme intermediates that cause disease symptoms. We hav...

journal_title：Molecular therapy. Nucleic acids

authors： Chan A,Liebow A,Yasuda M,Gan L,Racie T,Maier M,Kuchimanchi S,Foster D,Milstein S,Charisse K,Sehgal A,Manoharan M,Meyers R,Fitzgerald K,Simon A,Desnick RJ,Querbes W

更新日期：2015-11-03 00:00:00

abstract：:Long noncoding RNAs (lncRNAs), which serve as important and powerful regulators of various biological activities, have gained widespread attention in recent years. Emerging evidence has shown that some lncRNAs play important regulatory roles in osteoblast differentiation of mesenchymal stem cells (MSCs), suggesting a ...

journal_title：Molecular therapy. Nucleic acids

authors： Feng L,Shi L,Lu YF,Wang B,Tang T,Fu WM,He W,Li G,Zhang JF

更新日期：2018-06-01 00:00:00

abstract：:Besides the acquisition of pharmacokinetic parameters of antisense oligonucleotide microRNA (miRNA) inhibitors, such as measuring in vivo concentration, their pharmacodynamic characteristics are also of interest. An emerging and straightforward method for studying molecular interactions is microscale thermophoresis (M...

journal_title：Molecular therapy. Nucleic acids

authors： Herkt M,Batkai S,Thum T

更新日期：2019-12-06 00:00:00

abstract：:The anti-angiogenic and neurogenic pigment epithelium-derived factor (PEDF) demonstrated a potency to control choroidal neovascularization in age-related macular degeneration (AMD) patients. The goal of the present study was the development of an efficient and safe technique to integrate, ex vivo, the PEDF gene into r...

journal_title：Molecular therapy. Nucleic acids

authors： Pastor M,Johnen S,Harmening N,Quiviger M,Pailloux J,Kropp M,Walter P,Ivics Z,Izsvák Z,Thumann G,Scherman D,Marie C

更新日期：2018-06-01 00:00:00

abstract：:Spinocerebellar ataxia type 1 (SCA1) is a lethal, autosomal dominant neurodegenerative disease caused by a polyglutamine expansion in the ATAXIN-1 (ATXN1) protein. Preclinical studies demonstrate the therapeutic efficacy of approaches that target and reduce Atxn1 expression in a non-allele-specific manner. However, st...

journal_title：Molecular therapy. Nucleic acids

authors： O'Callaghan B,Hofstra B,Handler HP,Kordasiewicz HB,Cole T,Duvick L,Friedrich J,Rainwater O,Yang P,Benneyworth M,Nichols-Meade T,Heal W,Ter Haar R,Henzler C,Orr HT

更新日期：2020-09-04 00:00:00

abstract：:Exosomes mediate cell-cell crosstalk in cancer progression by transferring a variety of biomolecules, including long noncoding RNAs (lncRNAs). Long non-coding RNA urothelial carcinoma-associated (UCA1) is a well-known lncRNA associated with the development and progression of various cancers, including colorectal cance...

journal_title：Molecular therapy. Nucleic acids

authors： Luan Y,Li X,Luan Y,Zhao R,Li Y,Liu L,Hao Y,Oleg Vladimir B,Jia L

更新日期：2020-03-06 00:00:00

abstract：:Triple-negative breast cancer (TNBC) is an aggressive subtype of breast cancer that lacks expression of the three most common receptors present on other subtypes, leaving it unsusceptible to current targeted or hormonal therapies. In this study, we introduce an alternative treatment strategy for TNBC that exploits its...

journal_title：Molecular therapy. Nucleic acids

authors： Valcourt DM,Day ES

更新日期：2020-09-04 00:00:00

abstract：:Hepatocellular carcinoma (HCC) has high fatality rate and limited therapeutic options. Here, we propose a new anti-HCC approach with high cancer-selectivity and efficient anticancer effects, based on adenovirus-mediated Tetrahymena group I trans-splicing ribozymes specifically inducing targeted suicide gene activity t...

journal_title：Molecular therapy. Nucleic acids

authors： Han SR,Lee CH,Im JY,Kim JH,Kim JH,Kim SJ,Cho YW,Kim E,Kim Y,Ryu JH,Ju MH,Jeong JS,Lee SW

更新日期：2020-10-31 00:00:00

abstract：:Schistosoma japonicum eggs trapped in host liver secretes microRNA (miRNA)-containing extracellular vesicles (EVs) that can be transferred to host cells. Recent studies demonstrated that miRNAs derived from plants can modulate gene expression and phenotype of mammalian cells in a cross-kingdom manner. In this study, w...

journal_title：Molecular therapy. Nucleic acids

authors： Lin Y,Zhu S,Hu C,Wang J,Jiang P,Zhu L,Li Z,Wang S,Zhang Y,Xu X,Pan W

更新日期：2019-12-06 00:00:00

abstract：:We previously demonstrated that expression of Notch-1 is associated with poor prognosis in lung adenocarcinoma (LAD) patients. The aim of this study is to reveal whether Notch-1 was associated with Taxanes-resistant LAD and, the underlying mechanisms. We collected 39 patients of advanced LAD treated with Taxanes and f...

journal_title：Molecular therapy. Nucleic acids

authors： Huang J,Chen Y,Li J,Zhang K,Chen J,Chen D,Feng B,Song H,Feng J,Wang R,Chen L

更新日期：2016-10-11 00:00:00

abstract：:Inflammatory mediators play a key role in the pathogenesis of acute respiratory distress syndrome (ARDS). In this study, we aimed to explore the involvement of the Kcnq1 opposite strand/antisense transcript 1 (Kcnq1ot1)/miR-381-3p/E26 transformation-specific proto-oncogene 2 (ETS2) axis in inflammation of lipopolysacc...

journal_title：Molecular therapy. Nucleic acids

authors： Jiang X,Yu M,Zhu T,Lou L,Chen X,Li Q,Wei D,Sun R

更新日期：2020-03-06 00:00:00

abstract：:MicroRNAs (miRNAs or miRs) play crucial roles in biological and pathological processes. Some miRNAs also appear as promising biomarkers and therapeutic tools. However, the epitranscriptomic regulation of miRNAs is not yet fully elucidated in all of their fields of application. We report that adenosine methylation of m...

journal_title：Molecular therapy. Nucleic acids

authors： Briand J,Sérandour AA,Nadaradjane A,Bougras-Cartron G,Heymann D,Ory B,Vallette FM,Cartron PF

更新日期：2020-08-14 00:00:00

abstract：:Human spermatogonial stem cells (SSCs) could have significant applications in reproductive medicine and regenerative medicine because of their great plasticity. The fate determinations of human SSCs are mediated by epigenetic factors. However, nothing is known about the regulation of non-coding RNA on human SSCs. Here...

journal_title：Molecular therapy. Nucleic acids

authors： Zhou F,Yuan Q,Zhang W,Niu M,Fu H,Qiu Q,Mao G,Wang H,Wen L,Wang H,Lu M,Li Z,He Z

更新日期：2018-09-07 00:00:00

abstract：:Recent studies reported that DNA methylation was involved in retinal cell death. Methyl-CpG binding domain protein 2 (Mbd2) is one of the DNA methylation readers. Its role and mechanism of regulation remain unclear. The ischemia/reperfusion (I/R) model in mice primary culture retinal ganglion cells (RGCs) and Mbd2 kno...

journal_title：Molecular therapy. Nucleic acids

authors： Ge Y,Zhang R,Feng Y,Li H

更新日期：2020-03-06 00:00:00

abstract：:Base editing systems show their power in modeling and correcting the pathogenic mutations of genetic diseases. Previous studies have already demonstrated the editing efficiency of BE3-mediated C-to-T conversion in human embryos. However, the precision and efficiency of a recently developed adenine base editor (ABE), w...

journal_title：Molecular therapy. Nucleic acids

authors： Li G,Liu X,Huang S,Zeng Y,Yang G,Lu Z,Zhang Y,Ma X,Wang L,Huang X,Liu J

更新日期：2019-09-06 00:00:00

abstract：:Recent reports underscore the unparalleled potential of antisense-oligonucleotide (ASO)-based approaches to ameliorate various pathological conditions. However, in vivo studies validating the effectiveness of a short ASO (<10-mer) in the context of a human disease have not been performed. One disease with proven amena...

journal_title：Molecular therapy. Nucleic acids

authors： Keil JM,Seo J,Howell MD,Hsu WH,Singh RN,DiDonato CJ

更新日期：2014-07-08 00:00:00

abstract：:Thanks to their ability to recognize biomolecular targets with high affinity and specificity, nucleic acid aptamers are increasingly investigated as diagnostic and therapeutic tools, particularly when their targets are cell-surface receptors. Here, we investigate the relationship between the folding of an anti-mouse t...

journal_title：Molecular therapy. Nucleic acids

authors： Porciani D,Signore G,Marchetti L,Mereghetti P,Nifosì R,Beltram F

更新日期：2014-01-28 00:00:00

abstract：:Transcription factor Yin Yang 1 (YY1) is upregulated in multiple tumors and plays essential roles in tumor proliferation and metastasis. However, the function of YY1 in breast cancer stemness remains unclear. Herein, we found that YY1 expression was negatively correlated with the overall survival and relapse-free surv...

journal_title：Molecular therapy. Nucleic acids

authors： Guo Q,Wang T,Yang Y,Gao L,Zhao Q,Zhang W,Xi T,Zheng L

更新日期：2020-09-04 00:00:00

abstract：:Nonviral vector systems are used increasingly in gene targeting and gene transfer applications. The piggyBac transposon represents an alternative integrating vector for in vivo gene transfer. We hypothesized that this system could achieve persistent gene transfer to the liver when administered systemically. We report ...

journal_title：Molecular therapy. Nucleic acids

authors： Burnight ER,Staber JM,Korsakov P,Li X,Brett BT,Scheetz TE,Craig NL,McCray PB Jr

更新日期：2012-10-16 00:00:00

abstract：:Development of DNA aptamer screens that are both simple and informative can increase the success rate of DNA aptamer selection and induce greater adoption. High eIF4e levels contribute to malignancies, thus eIF4e presents itself as a valuable target for DNA aptamer-based inhibition screen. Here, we demonstrate a metho...

journal_title：Molecular therapy. Nucleic acids

authors： Guo WM,Kong KW,Brown CJ,Quah ST,Yeo HL,Hoon S,Seow Y

更新日期：2014-12-16 00:00:00

abstract：:Engineered nuclease-mediated gene targeting through homologous recombination (HR) in hematopoietic stem and progenitor cells (HSPCs) has the potential to treat a variety of genetic hematologic and immunologic disorders. Here, we identify critical parameters to reproducibly achieve high frequencies of RNA-guided (singl...

journal_title：Molecular therapy. Nucleic acids

authors： Charlesworth CT,Camarena J,Cromer MK,Vaidyanathan S,Bak RO,Carte JM,Potter J,Dever DP,Porteus MH

更新日期：2018-09-07 00:00:00

abstract：:We have reported that of the 10 most commonly used adeno-associated virus (AAV) serotype vectors, AAV6 is the most efficient in transducing primary human hematopoietic stem cells (HSCs) in vitro, as well as in vivo. More recently, polyvinyl alcohol (PVA), was reported to be a superior replacement for human serum album...

journal_title：Molecular therapy. Nucleic acids

authors： Yang H,Qing K,Keeler GD,Yin L,Mietzsch M,Ling C,Hoffman BE,Agbandje-McKenna M,Tan M,Wang W,Srivastava A

更新日期：2020-06-05 00:00:00

abstract：:Adeno-associated virus (AAV) is the most common vector for clinical gene therapy of the CNS. This popularity originates from a high safety record and the longevity of transgene expression in neurons. Nevertheless, clinical efficacy for CNS indications is lacking, and one reason for this is the relatively limited sprea...

journal_title：Molecular therapy. Nucleic acids

authors： Kanaan NM,Sellnow RC,Boye SL,Coberly B,Bennett A,Agbandje-McKenna M,Sortwell CE,Hauswirth WW,Boye SE,Manfredsson FP

更新日期：2017-09-15 00:00:00

abstract：:Targeted nucleases are influential instruments for intervening in genome revision with great accuracy. RNA-guided Cas9 nucleases produced from clustered regularly interspaced short palindromic repeats (CRISPR)-Cas systems have noticeably altered the means to modify the genomes of distinct organisms. They can be notabl...

journal_title：Molecular therapy. Nucleic acids

authors： Wang X,Huang X,Fang X,Zhang Y,Wang W

更新日期：2016-11-15 00:00:00

abstract：:We developed an efficient system for delivering short interfering RNA (siRNA) to the liver by using α-tocopherol conjugation. The α-tocopherol-conjugated siRNA was effective and safe for RNA interference-mediated gene silencing in vivo. In contrast, when the 13-mer LNA (locked nucleic acid)-DNA gapmer antisense oligon...

journal_title：Molecular therapy. Nucleic acids

authors： Nishina T,Numata J,Nishina K,Yoshida-Tanaka K,Nitta K,Piao W,Iwata R,Ito S,Kuwahara H,Wada T,Mizusawa H,Yokota T

更新日期：2015-01-13 00:00:00

abstract：:Next-generation sequencing technology has been commonly applied to detect mitochondrial DNA (mtDNA) mutations, which are reported to be strongly associated with cancers. However, several key challenges still exist regarding bioinformatics analysis of mtDNA sequencing data that greatly affect the detection accuracy of ...

journal_title：Molecular therapy. Nucleic acids

authors： Guo S,Zhou K,Yuan Q,Su L,Liu Y,Ji X,Gu X,Guo X,Xing J

更新日期：2020-11-11 00:00:00

abstract：:The popularization of genome-wide analyses and RNA sequencing led to the discovery that a large part of the human genome, while effectively transcribed, does not encode proteins. Long non-coding RNAs have emerged as critical regulators of gene expression in both normal and disease states. Studies of long non-coding RN...

journal_title：Molecular therapy. Nucleic acids

authors： Gomes CPC,Spencer H,Ford KL,Michel LYM,Baker AH,Emanueli C,Balligand JL,Devaux Y,Cardiolinc network.

更新日期：2017-09-15 00:00:00

abstract：:Circular RNA (circRNA) is a novel subclass of noncoding-RNA molecules that participate in development and progression of a variety of human diseases via sponging microRNAs (miRNAs), but the role of circRNAs in Hashimoto's thyroiditis (HT) has not been defined. In this study, peripheral blood samples from five patients...

journal_title：Molecular therapy. Nucleic acids

authors： Xiong S,Peng H,Ding X,Wang X,Wang L,Wu C,Wang S,Xu H,Liu Y

更新日期：2019-09-06 00:00:00

abstract：:Ischemia-reperfusion (I/R) injury is a major side effect of the reperfusion treatment of the ischemic heart. Few therapies are available for the effective prevention of this injury caused by the oxidative stress-induced cardiomyocyte apoptosis. Metformin was shown to have a potential cardiac protective effect and abil...

journal_title：Molecular therapy. Nucleic acids

authors： Zhang Y,Liu X,Zhang L,Li X,Zhou Z,Jiao L,Shao Y,Li M,Leng B,Zhou Y,Liu T,Liu Q,Shan H,Du Z

更新日期：2018-12-07 00:00:00

abstract：:The role of microglia in the pathophysiology of ischemic retinal diseases has been studied extensively. Exosomes from microglial cells exert protective effects during several nervous system diseases, but their roles in hypoxia-induced retinopathy remain unclear. In our study, exosomes derived from microglial cells wer...

journal_title：Molecular therapy. Nucleic acids

authors： Xu W,Wu Y,Hu Z,Sun L,Dou G,Zhang Z,Wang H,Guo C,Wang Y

更新日期：2019-06-07 00:00:00