Repair of Mybpc3 mRNA by 5'-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy.

abstract：:Methyltransferase-like 3 (METTL3) is the main enzyme for N6-methyladenosine (m6A)-based methylation of RNAs and it has been implicated in many biological and pathophysiological processes. In this study, we aimed to explore the potential involvement of METTL3 in osteoblast differentiation and decipher the underlying ce...

journal_title：Molecular therapy. Nucleic acids

authors： Yan G,Yuan Y,He M,Gong R,Lei H,Zhou H,Wang W,Du W,Ma T,Liu S,Xu Z,Gao M,Yu M,Bian Y,Pang P,Li X,Yu S,Yang F,Cai B,Yang L

更新日期：2020-03-06 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：:F-box and WD repeat domain-containing protein 7 (FBW7) has been documented to be implicated in nuclear factor κB (NF-κB) signaling and inflammation, but its role in the pathogenesis of inflammatory bowel disease (IBD) remains unknown. FBW7 was increased both in colon tissues from IBD patients and trinitrobenzene sulph...

journal_title：Molecular therapy. Nucleic acids

authors： Meng Q,Wu W,Pei T,Xue J,Xiao P,Sun L,Li L,Liang D

更新日期：2020-03-06 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：:Recent studies have revealed the cytoprotective roles of microRNAs (miRNAs) miR-21 and miR-146a against ischemic cardiac injuries. While these studies investigated each of these miRNAs as an independent individual factor, our previous study has suggested the possible interaction between these two miRNAs. The present s...

journal_title：Molecular therapy. Nucleic acids

authors： Huang W,Tian SS,Hang PZ,Sun C,Guo J,Du ZM

更新日期：2016-03-15 00:00:00

abstract：:A gene therapeutic platform needs to be both efficient and safe. The criterion of safety is particularly important for diseases like hepatocellular carcinoma (HCC), which develop in a background of an already compromised liver. Gene vectors can be constructed either by targeting HCC or by detargeting liver and/or othe...

journal_title：Molecular therapy. Nucleic acids

authors： Dhungel B,Ramlogan-Steel CA,Layton CJ,Steel JC

更新日期：2018-12-07 00:00:00

abstract：:A hexanucleotide GGGGCC expansion in intron 1 of chromosome 9 open reading frame 72 (C9orf72) gene is the most frequent cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The corresponding repeat-containing sense and antisense transcripts cause a gain of toxicity through the accumulation o...

journal_title：Molecular therapy. Nucleic acids

authors： Martier R,Liefhebber JM,García-Osta A,Miniarikova J,Cuadrado-Tejedor M,Espelosin M,Ursua S,Petry H,van Deventer SJ,Evers MM,Konstantinova P

更新日期：2019-06-07 00:00:00

abstract：:Polycystic ovary syndrome (PCOS), characterized by the dysfunction of endocrine metabolism, is a common disease among women. Insulin (INS) resistance (IR) is considered as an obstruction to effective PCOS treatment. Here, we aimed to explore the mechanism by which microRNA-222 (miR-222) affects IR in PCOS via Pten. Qu...

journal_title：Molecular therapy. Nucleic acids

authors： Ye H,Liu XJ,Hui Y,Liang YH,Li CH,Wan Q

更新日期：2020-07-10 00:00:00

abstract：:Recent studies have suggested that microRNA let-7i is a tumor suppressor in human cancers, including esophageal cancer, but its underlying mechanism is not yet fully understood. We investigated the role and mechanisms of let-7i in the progression of esophageal cancer. We first showed that let-7i was downregulated in e...

journal_title：Molecular therapy. Nucleic acids

authors： Yang Y,Li W,Wei B,Wu K,Liu D,Zhu D,Zhang C,Wen F,Fan Y,Zhao S

更新日期：2020-09-16 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：:Cardiac hypertrophy accompanied by maladaptive cardiac remodeling is the uppermost risk factor for the development of heart failure. Long non-coding RNAs (lncRNAs) and microRNAs (miRNAs) have various biological functions, and their vital role in the regulation of cardiac hypertrophy still needs to be explored. In this...

journal_title：Molecular therapy. Nucleic acids

authors： Lv L,Li T,Li X,Xu C,Liu Q,Jiang H,Li Y,Liu Y,Yan H,Huang Q,Zhou Y,Zhang M,Shan H,Liang H

更新日期：2018-03-02 00:00:00

abstract：:Apoptosis and calcification of endplate chondrocytes (EPCs) can exacerbate intervertebral disc degeneration (IVDD). Mesenchymal stem cell-derived exosomes (MSC-exosomes) are reported to have the therapeutic potential in IVDD. However, the effects and related mechanisms of MSC-exosomes on EPCs are still unclear. We aim...

journal_title：Molecular therapy. Nucleic acids

authors： Xie L,Chen Z,Liu M,Huang W,Zou F,Ma X,Tao J,Guo J,Xia X,Lyu F,Wang H,Zheng C,Jiang J

更新日期：2020-09-26 00:00:00

abstract：:Acute lung injury (ALI) is a pulmonary disorder that causes acute respiratory failure, thus leading to relative high mortality worldwide. However, the molecular mechanisms of ALI remain largely unknown. MicroRNA (miRNA)-dependent control of gene expression at a post-transcriptional level has been recently reported. He...

journal_title：Molecular therapy. Nucleic acids

authors： Ying Y,Mao Y,Yao M

更新日期：2019-12-06 00:00:00

abstract：:Neovascular age-related macular degeneration (AMD) is characterized by the formation of choroidal neovascularization, which is responsible for more than 80% of cases of severe vision loss. Ubiquitin protein ligase E3D (UBE3D) gene missense has been proven to be associated with neovascular AMD in the East Asian populat...

journal_title：Molecular therapy. Nucleic acids

authors： Xia H,Zhang Q,Shen Y,Bai Y,Ma X,Zhang B,Qi Y,Zhang J,Hu Q,Du W,Zhu L,Zhou P,Wang B,Xu H,Huang L,Li X

更新日期：2020-06-05 00:00:00

abstract：:Circular RNAs (circRNAs) were recently reported to be involved in the pathogenesis of ovarian cancer (OC); however, the molecular mechanisms of circRNAs in tumor progression and paclitaxel (PTX) resistance of OC remain largely undetermined. Here, we focused on circTNPO3 (hsa_circ_0001741), which is located on chromoso...

journal_title：Molecular therapy. Nucleic acids

authors： Xia B,Zhao Z,Wu Y,Wang Y,Zhao Y,Wang J

更新日期：2020-09-04 00:00:00

abstract：:Long noncoding RNA (lncRNA) has been implicated in cancer, but little is known about the role of lncRNAs as regulators of tumor metastasis. In the present study, we demonstrate that lncRNA TRERNA1 acts like an enhancer of SNAI1 to promote cell invasion and migration and to contribute to metastasis of gastric cancer (G...

journal_title：Molecular therapy. Nucleic acids

authors： Wu H,Hu Y,Liu X,Song W,Gong P,Zhang K,Chen Z,Zhou M,Shen X,Qian Y,Fan H

更新日期：2017-09-15 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：:The ability to selectively target disease-related tissues with molecules is critical to the design of effective therapeutic and diagnostic reagents. Recognizing the differences between the in vivo environment and in vitro conditions, we employed an in vivo selection strategy to identify RNA aptamers (targeting motifs)...

journal_title：Molecular therapy. Nucleic acids

authors： Mi J,Ray P,Liu J,Kuan CT,Xu J,Hsu D,Sullenger BA,White RR,Clary BM

更新日期：2016-04-26 00:00:00

abstract：:With the development of science and biotechnology, many evidences show that ncRNAs play an important role in the development of important biological processes, especially in chromatin modification, cell differentiation and proliferation, RNA progressing, human diseases, etc. Moreover, lncRNAs account for the majority ...

journal_title：Molecular therapy. Nucleic acids

authors： Zhao Q,Yu H,Ming Z,Hu H,Ren G,Liu H

更新日期：2018-12-07 00:00:00

abstract：:[This corrects the article DOI: 10.1038/mtna.2016.46.]. ...

journal_title：Molecular therapy. Nucleic acids

authors： Cao L,Han G,Lin C,Gu B,Gao X,Moulton HM,Seow Y,Yin H

更新日期：2020-11-19 00:00:00

abstract：:MicroRNAs (miRNAs) are an important class of small noncoding RNA molecules that serve as excellent biomarkers of various diseases. However, current miRNA biomarkers, including those comprised of multiple miRNAs, work at a single-miRNA level but not at a miRNA-set level, which is defined as a group of miRNAs sharing co...

journal_title：Molecular therapy. Nucleic acids

authors： Shi J,Cui Q

更新日期：2020-09-04 00:00:00

abstract：:A number of studies have reported that aberrant expression of microRNAs (miRNAs) closely correlates with the bone metastasis of prostate cancer (PCa). However, clinical significance and functional roles of both strands of a single miRNA in bone metastasis of PCa remain undefined. Here, we reported that miR-582-3p and ...

journal_title：Molecular therapy. Nucleic acids

authors： Huang S,Zou C,Tang Y,Wa Q,Peng X,Chen X,Yang C,Ren D,Huang Y,Liao Z,Huang S,Zou X,Pan J

更新日期：2019-06-07 00:00:00

abstract：:Cutaneous squamous cell carcinoma (CSCC) is a malignant proliferation of cutaneous epithelium that has been observed to have an alarming rise in incidence. Numerous studies have demonstrated microRNAs (miRNAs or miRs) as important biomarkers in the diagnosis, prognosis, and treatment of CSCC. This study aims to invest...

journal_title：Molecular therapy. Nucleic acids

authors： Ting W,Feng C,Zhang M,Long F,Bai M

更新日期：2020-09-04 00:00:00

abstract：:MicroRNAs (miRNAs) are a class of genome-encoded small RNAs that are primarily considered to be post-transcriptional negative regulators of gene expression acting in the cytoplasm. Over a decade of research has focused on this canonical paradigm of miRNA function, with many success stories. Indeed, miRNAs have been id...

journal_title：Molecular therapy. Nucleic acids

authors： Roberts TC

更新日期：2014-08-19 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：:Antisense oligonucleotides complementary to RNA targets promise generality and ease of drug design. The first systemically administered antisense drug was recently approved for treatment and others are in clinical development. Chemical modifications that increase the hybridization affinity of oligonucleotides are reas...

journal_title：Molecular therapy. Nucleic acids

authors： Pedersen L,Hagedorn PH,Lindholm MW,Lindow M

更新日期：2014-02-18 00:00:00

abstract：:PTEN expression is lost in many cancers, and even small changes in PTEN activity affect susceptibility and prognosis in a range of highly aggressive malignancies, such as melanoma and triple-negative breast cancer (TNBC). Loss of PTEN expression occurs via multiple mechanisms, including mutation, transcriptional repre...

journal_title：Molecular therapy. Nucleic acids

authors： Moses C,Nugent F,Waryah CB,Garcia-Bloj B,Harvey AR,Blancafort P

更新日期：2019-03-01 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：:The microRNA(miRNA)-34a is a key regulator of tumor suppression. It controls the expression of a plethora of target proteins involved in cell cycle, differentiation and apoptosis, and antagonizes processes that are necessary for basic cancer cell viability as well as cancer stemness, metastasis, and chemoresistance. I...

journal_title：Molecular therapy. Nucleic acids

authors： Misso G,Di Martino MT,De Rosa G,Farooqi AA,Lombardi A,Campani V,Zarone MR,Gullà A,Tagliaferri P,Tassone P,Caraglia M

更新日期：2014-09-23 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