Mathematical Modeling: A Tool for Optimization of Lipid Nanoparticle-Mediated Delivery of siRNA.

abstract：:Although patients with non-small cell lung cancer harboring activating mutations in the epidermal growth factor receptor (EGFR) show good clinical response to EGFR tyrosine kinase inhibitors (TKIs), patients eventually develop acquired resistance. Previous studies have shown that several microRNAs (miRNAs) are involve...

journal_title：Molecular therapy. Nucleic acids

authors： Liu YN,Tsai MF,Wu SG,Chang TH,Tsai TH,Gow CH,Wang HY,Shih JY

更新日期：2020-09-16 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：:Effective clinical application of antiviral immunotherapies necessitates enhancing the functional state of natural killer (NK) and CD8(+) T cells. An important mechanism for the establishment of viral persistence in the liver is the activation of the PD-1/PD-L1 inhibitory pathway. To examine the role of hepatic myeloi...

journal_title：Molecular therapy. Nucleic acids

authors： Dolina JS,Sung SS,Novobrantseva TI,Nguyen TM,Hahn YS

更新日期：2013-02-19 00:00:00

abstract：:N4-acetylcytidine (ac4C) is often considered to be a conservative, chemically modified nucleoside present on tRNA and rRNA. Recent studies have shown extensive ac4C modifications in human and yeast mRNAs. ac4C helps to correctly read codons during translation and improves translation efficiency and the stability of mR...

journal_title：Molecular therapy. Nucleic acids

authors： Jin G,Xu M,Zou M,Duan S

更新日期：2020-06-05 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：:Vascular endothelial growth factors (VEGFs) are hypoxia-inducible secreted proteins to promote angiogenesis, in which VEGF-A is an important molecule that binds and activates VEGF receptor-1 (VEGFR-1) and VEGFR-2. In this study, two DNA aptamers, Apt01 and Apt02, were successfully isolated by alternating consecutive s...

journal_title：Molecular therapy. Nucleic acids

authors： Yoshitomi T,Hayashi M,Oguro T,Kimura K,Wayama F,Furusho H,Yoshimoto K

更新日期：2020-03-06 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：: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：:Alzheimer's disease (AD) is a multifactorial, fatal neurodegenerative disorder characterized by the abnormal accumulation of Aβ and Tau deposits in the brain. There is no cure for AD, and failure at different clinical trials emphasizes the need for new treatments. In recent years, significant progress has been made to...

journal_title：Molecular therapy. Nucleic acids

authors： Parsi S,Smith PY,Goupil C,Dorval V,Hébert SS

更新日期：2015-10-06 00:00:00

abstract：:PCTAIRE1/CDK16/PCTK1 plays critical roles in cancer cell proliferation and antiapoptosis. To advance our previously published in vitro results with PCTAIRE1 silencing, we examined the in vivo therapeutic potential of this approach by using small interfering RNA (siRNA) encapsulated by lipid nanoparticles. Therapy expe...

journal_title：Molecular therapy. Nucleic acids

authors： Yanagi T,Tachikawa K,Wilkie-Grantham R,Hishiki A,Nagai K,Toyonaga E,Chivukula P,Matsuzawa S

更新日期：2016-06-28 00:00:00

abstract：:The transforming growth factor-β (TGF-β)/Smads signal plays an important role in cancer metastasis by mediating the epithelial-mesenchymal transition (EMT) in cancer cells. lnc-TSI is a recently identified long noncoding RNA that negatively regulates the TGF-β/Smads signal. The present study was conducted to test the ...

journal_title：Molecular therapy. Nucleic acids

authors： Wang P,Chen W,Ma T,Lin Z,Liu C,Liu Y,Hou FF

更新日期：2020-08-05 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：:Retinoic acid-inducible gene-I (RIG-I) is a cytosolic pathogen sensor that is crucial against a number of viral infections. Many viruses have evolved to inhibit pathogen sensors to suppress host innate immune responses. In the case of influenza, nonstructural protein 1 (NS1) suppresses RIG-I function, leading to viral...

journal_title：Molecular therapy. Nucleic acids

authors： Singh N,Ranjan P,Cao W,Patel J,Gangappa S,Davidson BA,Sullivan JM,Prasad PN,Knight PR,Sambhara S

更新日期：2020-03-06 00:00:00

abstract：:Following tendon injury, the development of fibrotic healing response impairs tendon function and restricts tendon motion. Peritendinous tissue fibrosis poses a major clinical problem in hand surgery. Communication between macrophages and tendon cells has a critical role in regulating the tendon-healing process. Yet, ...

journal_title：Molecular therapy. Nucleic acids

authors： Cui H,He Y,Chen S,Zhang D,Yu Y,Fan C

更新日期：2019-03-01 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：: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：:miR-221 is overexpressed in several malignancies where it promotes tumor growth and survival by interfering with gene transcripts, including p27Kip1, PUMA, PTEN, and p57Kip2. We previously demonstrated that a novel 13-mer miR-221 inhibitor (locked nucleic acid [LNA]-i-miR-221) exerts antitumor activity against human c...

journal_title：Molecular therapy. Nucleic acids

authors： Di Martino MT,Arbitrio M,Caracciolo D,Scionti F,Tagliaferri P,Tassone P

更新日期：2020-06-05 00:00:00

abstract：:Neuroblastoma is the primary cause of cancer death in childhood. METTL14 is tightly linked to cancer. However, whether single-nucleotide polymorphisms (SNPs) in the METTL14 gene could predispose to neuroblastoma susceptibility lacks evidence. With an epidemiology case-control study, associations between METTL14 gene S...

journal_title：Molecular therapy. Nucleic acids

authors： Zhuo Z,Lu H,Zhu J,Hua RX,Li Y,Yang Z,Zhang J,Cheng J,Zhou H,Li S,Li L,Xia H,He J

更新日期：2020-08-14 00:00:00

abstract：:Schizophrenia (SCZ) is a devastating genetic mental disorder. Identification of the SCZ risk genes in brains is helpful to understand this disease. Thus, we first used the minimum Redundancy-Maximum Relevance (mRMR) approach to integrate the genome-wide sequence analysis results on SCZ and the expression quantitative ...

journal_title：Molecular therapy. Nucleic acids

authors： Cai L,Huang T,Su J,Zhang X,Chen W,Zhang F,He L,Chou KC

更新日期：2018-09-07 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：:The human proinsulin gene (INS) contains a thymine-to-adenine variant (rs689) located in the 3' splice site (3' ss) recognition motif of the first intron. The adenine at rs689 is strongly associated with type 1 diabetes. By weakening the polypyrimidine tract, the adenine allele reduces the efficiency of intron 1 splic...

journal_title：Molecular therapy. Nucleic acids

authors： Lages A,Proud CG,Holloway JW,Vorechovsky I

更新日期：2019-06-07 00:00:00

abstract：:As the human lifespan has increased due to developments in medical technology, the number of patients with neurological diseases has rapidly increased. Therefore, studies on effective treatments for neurological diseases are becoming increasingly important. To perform these studies, it is essential to obtain a large n...

journal_title：Molecular therapy. Nucleic acids

authors： Daekee K,Mi-Jung H,Minjun J,Hee-Jin A,Kwang-Won S,Kyung-Sun K

更新日期：2019-03-01 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：:Septic shock with heart dysfunction is very common in intensive care units. However, whether long noncoding RNA (lncRNA) and mRNA profiles differ between patients with and without myocardial depression is unknown. We generated rat models of hypodynamic septic shock induced by lipopolysaccharide. A total of 12 rat mode...

journal_title：Molecular therapy. Nucleic acids

authors： Zhang TN,Goodwin JE,Liu B,Li D,Wen R,Yang N,Xia J,Zhou H,Zhang T,Song WL,Liu CF

更新日期：2019-09-06 00:00:00

abstract：:Antisense oligonucleotides (AOs) are currently the most promising therapeutic intervention for Duchenne muscular dystrophy (DMD). AOs modulate dystrophin pre-mRNA splicing, thereby specifically restoring the dystrophin reading frame and generating a truncated but semifunctional dystrophin protein. Challenges in the de...

journal_title：Molecular therapy. Nucleic acids

authors： Betts C,Saleh AF,Arzumanov AA,Hammond SM,Godfrey C,Coursindel T,Gait MJ,Wood MJ

更新日期：2012-08-14 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：:Oral squamous cell carcinoma (OSCC) is a highly recurrent form of cancer arising from the oral epithelium, which is the result of mutational change due to etiological factors such as tobacco, smoking, chewing of areca nuts, and alcohol consumption. OSCC occurrence has been observed to be prevalent in different regions...

journal_title：Molecular therapy. Nucleic acids

authors： Saikishore R,Velmurugan P,Ranjithkumar D,Latha R,Sathiamoorthi T,Arun A,Ravi AV,Sivakumar S

更新日期：2020-09-06 00:00:00

abstract：:Fabry disease is a rare X-linked lysosomal storage disorder caused by deficiency of the α-galactosidase A (α-Gal A) enzyme, which is encoded by the GLA gene. GLA transcription in humans produces a major mRNA encoding α-Gal A and a minor mRNA of unknown function, which retains a 57-nucleotide-long cryptic exon between ...

journal_title：Molecular therapy. Nucleic acids

authors： Ferri L,Covello G,Caciotti A,Guerrini R,Denti MA,Morrone A

更新日期：2016-10-25 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：:Nonviral gene delivery is a promising therapeutic approach because of its safety and controllability, yet limited gene transfer efficacy is a common issue. Most nonviral strategies rely upon endosomal escape designs; however, endosomal escape is often uncorrelated with improved gene transfer and membranolytic structur...

journal_title：Molecular therapy. Nucleic acids

authors： Ross NL,Munsell EV,Sabanayagam C,Sullivan MO

更新日期：2015-02-10 00:00:00