Adeno-associated Virus-mediated, Mifepristone-regulated Transgene Expression in the Brain.

abstract：:Upregulation of long non-coding RNA LINC00963 has been observed in several cancer types. In this study, we analyzed the clinical and biological significance of LINC00963 in breast cancer. The key microRNA (miR) mediating the action of LINC00963 was identified. We show that LINC00963 upregulation is correlated with agg...

journal_title：Molecular therapy. Nucleic acids

authors： Zhang N,Zeng X,Sun C,Guo H,Wang T,Wei L,Zhang Y,Zhao J,Ma X

更新日期：2019-12-06 00:00:00

abstract：:RNA trans-splicing has been explored as a therapeutic option for a variety of genetic diseases, but not for cardiac genetic disease. Hypertrophic cardiomyopathy (HCM) is an autosomal-dominant disease, characterized by left ventricular hypertrophy (LVH) and diastolic dysfunction. MYBPC3, encoding cardiac myosin-binding...

journal_title：Molecular therapy. Nucleic acids

authors： Mearini G,Stimpel D,Krämer E,Geertz B,Braren I,Gedicke-Hornung C,Précigout G,Müller OJ,Katus HA,Eschenhagen T,Voit T,Garcia L,Lorain S,Carrier L

更新日期：2013-07-02 00:00:00

abstract：:Novel nanoscale bioconjugates combining unique plasmonic photothermal properties of gold nanoparticles (AuNPs) with targeted delivery using cell-specific DNA aptamers have a tremendous potential for medical diagnostics and therapy of many cell-based diseases. In this study, we demonstrate the high anti-cancer activity...

journal_title：Molecular therapy. Nucleic acids

authors： Kolovskaya OS,Zamay TN,Belyanina IV,Karlova E,Garanzha I,Aleksandrovsky AS,Kirichenko A,Dubynina AV,Sokolov AE,Zamay GS,Glazyrin YE,Zamay S,Ivanchenko T,Chanchikova N,Tokarev N,Shepelevich N,Ozerskaya A,Badrin E,Belug

更新日期：2017-12-15 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：:The current antibody-mediated numeration assays of circulating tumor cells (CTCs) require multiple steps and are time-consuming. To overcome these technical limitations, a cancer cell-activatable aptamer-reporter was formulated by conjugating a biomarker-specific aptamer sequence with paired fluorochrome-quencher mole...

journal_title：Molecular therapy. Nucleic acids

authors： Zeng Z,Tung CH,Zu Y

更新日期：2014-08-12 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：:The fluorescent quantum dots (QDs) delivered small interfering RNAs (siRNAs) targeting β-secretase (BACE1) to achieve high transfection efficiency of siRNAs and reduction of β-amyloid (Aβ) in nerve cells. The CdSe/ZnS QDs with the conjugation of amino-polyethylene glycol (PEG) were synthesized. Negatively charged siRN...

journal_title：Molecular therapy. Nucleic acids

authors： Li S,Liu Z,Ji F,Xiao Z,Wang M,Peng Y,Zhang Y,Liu L,Liang Z,Li F

更新日期：2012-04-24 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：:Current acute myeloid leukemia (AML) disease models face severe limitations because most of them induce un-physiological gene expressions that do not represent conditions in AML patients and/or depend on external promoters for regulation of gene expression/repression. Furthermore, many AML models are based on reciproc...

journal_title：Molecular therapy. Nucleic acids

authors： Brabetz O,Alla V,Angenendt L,Schliemann C,Berdel WE,Arteaga MF,Mikesch JH

更新日期：2017-03-17 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：:We previously found that inhibition of p66Shc confers protection against hepatic stellate cell (HSC) activation during liver fibrosis. However, the effect of p66Shc on HSC proliferation, as well as the mechanism by which p66Shc is modulated, remains unknown. Here, we elucidated the effect of p66Shc on HSC proliferatio...

journal_title：Molecular therapy. Nucleic acids

authors： Wang Z,Zhao Y,Zhao H,Zhou J,Feng D,Tang F,Li Y,Lv L,Chen Z,Ma X,Tian X,Yao J

更新日期：2020-09-04 00:00:00

abstract：:As a diverse and abundant class of endogenous RNAs, circular RNAs (circRNAs) participate in processes including cell proliferation and apoptosis. Nevertheless, few researchers have investigated the function of circRNAs in bovine muscle development. Based on existing sequencing data, we identified circINSR. The localiz...

journal_title：Molecular therapy. Nucleic acids

authors： Shen X,Zhang X,Ru W,Huang Y,Lan X,Lei C,Chen H

更新日期：2020-03-06 00:00:00

abstract：:MicroRNAs (miRNAs) are small noncoding RNAs approximately 20-25 nt in length, which play crucial roles through directly binding to corresponding 3' UTR of targeted mRNAs. It has been reported that miRNAs are involved in numerous of diseases, including cancers. Recently, miR-134 has been identified to dysregulate in ha...

journal_title：Molecular therapy. Nucleic acids

authors： Pan JY,Zhang F,Sun CC,Li SJ,Li G,Gong FY,Bo T,He J,Hua RX,Hu WD,Yuan ZP,Wang X,He QQ,Li DJ

更新日期：2017-03-17 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：: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：:Leukemias arise from genetic alterations in normal hematopoietic stem or progenitor cells, leading to impaired regulation of proliferation, differentiation, apoptosis, and survival of the transformed cells. With the advent of RNA interference (RNAi) and the short interfering RNA (siRNA) as its pharmacological mediator...

journal_title：Molecular therapy. Nucleic acids

authors： Landry B,Valencia-Serna J,Gul-Uludag H,Jiang X,Janowska-Wieczorek A,Brandwein J,Uludag H

更新日期：2015-05-12 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：:Abnormal synthesis of extracellular matrix (ECM), especially collagen type IV (Col IV), in human retinal capillary endothelial cells (HRCECs) and resultant basement membrane (BM) thickening is the most prominent and characteristic feature of early diabetic retinopathy (DR). Osteopontin (OPN) has been shown to play an ...

journal_title：Molecular therapy. Nucleic acids

authors： Duan P,Chen S,Zeng Y,Xu H,Liu Y

更新日期：2020-06-05 00:00:00

abstract：:Monogenic disorders (MGDs), which are caused by single gene mutations, have a serious effect on human health. Among these, β-thalassemia (β-thal) represents one of the most common hereditary hematological diseases caused by mutations in the human hemoglobin β (HBB) gene. The technologies of induced pluripotent stem ce...

journal_title：Molecular therapy. Nucleic acids

authors： Liu Y,Yang Y,Kang X,Lin B,Yu Q,Song B,Gao G,Chen Y,Sun X,Li X,Bu L,Fan Y

更新日期：2017-03-17 00:00:00

abstract：:Bioluminescence imaging (BLI) has shown to be crucial for monitoring in vivo biological processes. So far, only dual bioluminescence imaging using firefly (Fluc) and Renilla or Gaussia (Gluc) luciferase has been achieved due to the lack of availability of other efficiently expressed luciferases using different substra...

journal_title：Molecular therapy. Nucleic acids

authors： Maguire CA,Bovenberg MS,Crommentuijn MH,Niers JM,Kerami M,Teng J,Sena-Esteves M,Badr CE,Tannous BA

更新日期：2013-06-18 00:00:00

abstract：:Gastric cancer (GC) is one of the leading causes of cancer-related deaths worldwide. Accumulating evidence reveals the significance of long non-coding RNAs (lncRNAs) in various cancers. The current study aimed to evaluate the role of GATA6 antisense RNA 1 (GATA6-AS1) in the epithelial-mesenchymal transition (EMT) and ...

journal_title：Molecular therapy. Nucleic acids

authors： Li ZT,Zhang X,Wang DW,Xu J,Kou KJ,Wang ZW,Yong G,Liang DS,Sun XY

更新日期：2020-03-06 00:00:00

abstract：:The melanoma cell adhesion molecule (MCAM) is involved in melanoma development and its progression, including invasiveness, metastatic potential and angiogenesis. Therefore, MCAM represents a potential target for gene therapy of melanoma, whose expression could be hindered with posttranscriptional specific gene silenc...

journal_title：Molecular therapy. Nucleic acids

authors： Prosen L,Markelc B,Dolinsek T,Music B,Cemazar M,Sersa G

更新日期：2014-10-28 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：:Ribozyme genes were designed to reduce voluntary alcohol drinking in a rat model of alcohol dependence. Acetaldehyde generated from alcohol in the liver is metabolized by the mitochondrial aldehyde dehydrogenase (ALDH2) such that diminishing ALDH2 activity leads to the aversive effects of blood acetaldehyde upon alcoh...

journal_title：Molecular therapy. Nucleic acids

authors： Sapag A,Irrazábal T,Lobos-González L,Muñoz-Brauning CR,Quintanilla ME,Tampier L

更新日期：2016-07-12 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：:Circular RNAs (circRNAs) represent a class of noncoding RNAs with a wide expression pattern, and they constitute an important layer of the genome regulatory network. To date, the expression pattern and regulatory potency of circRNAs in the retina, a key part of the central nervous system, are not yet well understood. ...

journal_title：Molecular therapy. Nucleic acids

authors： Chen XJ,Zhang ZC,Wang XY,Zhao HQ,Li ML,Ma Y,Ji YY,Zhang CJ,Wu KC,Xiang L,Sun LF,Zhou M,Jin ZB

更新日期：2020-03-06 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 have shown that zinc finger nucleases (ZFNs) are powerful reagents for making site-specific genomic modifications. The generic structure of these enzymes includes a ZF DNA-binding domain and nuclease domain (Fn) are separated by an amino acid "linker" and cut genomic DNA at sites that have a generic str...

journal_title：Molecular therapy. Nucleic acids

authors： Wilson KA,McEwen AE,Pruett-Miller SM,Zhang J,Kildebeck EJ,Porteus MH

更新日期：2013-04-30 00:00:00

abstract：:Pancreatic cancer is characterized by chemoresistance after several cycles of chemotherapy, which is a major issue responsible for treatment failure of pancreatic cancer. Therefore, it is necessary to explore the specific mechanism underlying chemotherapeutic resistance to overcome this issue. Here we report that miR-...

journal_title：Molecular therapy. Nucleic acids

authors： Zhang X,Ren D,Wu X,Lin X,Ye L,Lin C,Wu S,Zhu J,Peng X,Song L

更新日期：2018-06-01 00:00:00

abstract：:External guide sequence (EGS) RNAs are associated with ribonuclease P (RNase P), a tRNA processing enzyme, and represent promising agents for gene-targeting applications as they can direct RNase-P-mediated cleavage of a target mRNA. Using murine cytomegalovirus (MCMV) as a model system, we examined the antiviral effec...

journal_title：Molecular therapy. Nucleic acids

authors： Li W,Sheng J,Xu M,Vu GP,Yang Z,Liu Y,Sun X,Trang P,Lu S,Liu F

更新日期：2017-12-15 00:00:00