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：:Long noncoding RNAs (lncRNAs) have been implicated in cancer biogenesis and prognosis. However, we still lack knowledge on their function during glioma progression. In this study, we analyzed the lncRNA expression profile across 907 glioma patients in grades II, III, and IV. Widespread dynamic expression of lncRNAs du...

journal_title：Molecular therapy. Nucleic acids

authors： Lin X,Jiang T,Bai J,Li J,Wang T,Xiao J,Tian Y,Jin X,Shao T,Xu J,Chen L,Wang L,Li Y

更新日期：2018-12-07 00:00:00

abstract：:As key regulators of gene expression, microRNAs (miRNAs) have emerged as targets in basic experimentation and therapy. Administration of DNA-encoded RNA molecules, targeting miRNAs through base pairing, is one viable strategy for inhibiting specific miRNAs. A naturally occurring circular RNA (circRNA), ciRS-7, serving...

journal_title：Molecular therapy. Nucleic acids

authors： Hollensen AK,Andersen S,Hjorth K,Bak RO,Hansen TB,Kjems J,Aagaard L,Damgaard CK,Mikkelsen JG

更新日期：2018-12-07 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：: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：:Non-coding RNAs play an important role in the pathogenesis of pulmonary arterial hypertension (PAH). The aim of this study was to characterize the therapeutic role of melatonin as well as the underlying molecular mechanism (its effects on the expression of H19 and its downstream signaling pathways) in the treatment of...

journal_title：Molecular therapy. Nucleic acids

authors： Wang R,Zhou S,Wu P,Li M,Ding X,Sun L,Xu X,Zhou X,Zhou L,Cao C,Fei G

更新日期：2018-12-07 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：:Spermatogonial stem cells (SSCs) have significant applications in reproductive and regenerative medicine. However, nothing is known about genes in mediating human SSCs. Here we have explored for the first time the function and mechanism of P21-activated kinase 1 (PAK1) in regulating the proliferation and apoptosis of ...

journal_title：Molecular therapy. Nucleic acids

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

更新日期：2018-09-07 00:00:00

abstract：:Breast cancer, as the most common cancer in women worldwide, represents about 30% of all cancers affecting women. Long non-coding RNAs (lncRNAs) have been implicated in the regulation of several biological processes, and their dysregulation in cancer has well been documented. To investigate possible age-dependent vari...

journal_title：Molecular therapy. Nucleic acids

authors： Arshi A,Sharifi FS,Khorramian Ghahfarokhi M,Faghih Z,Doosti A,Ostovari S,Mahmoudi Maymand E,Ghahramani Seno MM

更新日期：2018-09-07 00:00:00

abstract：:Aberrant insulin-like growth factor I receptor (IGF1R) signaling pathway serves as a well-established target for cancer drug therapy. The intragenic antisense long noncoding RNA (lncRNA) IRAIN, a putative tumor suppressor, is downregulated in breast cancer cells, while IGF1R is overexpressed, leading to an abnormal IG...

journal_title：Molecular therapy. Nucleic acids

authors： Pian L,Wen X,Kang L,Li Z,Nie Y,Du Z,Yu D,Zhou L,Jia L,Chen N,Li D,Zhang S,Li W,Hoffman AR,Sun J,Cui J,Hu JF

更新日期：2018-09-07 00:00:00

abstract：:Clinical translation of small interfering RNA (siRNA) nanocarriers is hindered by limited knowledge regarding the parameters that regulate interactions between nanocarriers and biological systems. To address this, we investigated the influence of polycation-based nanocarrier architecture on intracellular siRNA deliver...

journal_title：Molecular therapy. Nucleic acids

authors： Melamed JR,Kreuzberger NL,Goyal R,Day ES

更新日期：2018-09-07 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：: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：:Gemcitabine has been considered a first-line chemotherapy agent for the treatment of pancreatic cancer. However, the initial response rate of gemcitabine is low and chemoresistance occurs frequently. Aptamers can be effectively internalized into cancer cells via binding to target molecules with high affinity and speci...

journal_title：Molecular therapy. Nucleic acids

authors： Park JY,Cho YL,Chae JR,Moon SH,Cho WG,Choi YJ,Lee SJ,Kang WJ

更新日期：2018-09-07 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：: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：:Continuous innovation of revolutionizing genome engineering technologies calls for an intensified focus on new delivery technologies that not only match the inventiveness of genome editors but also enable the combination of potent delivery and time-restricted action of genome-modifying bits and tools. We have previous...

journal_title：Molecular therapy. Nucleic acids

authors： Skipper KA,Nielsen MG,Andersen S,Ryø LB,Bak RO,Mikkelsen JG

更新日期：2018-06-01 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：:Epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) are used clinically as target therapies for lung cancer patients, but the occurrence of acquired drug resistance limits their efficacy. Nicotinamide N-methyltransferase (NNMT), a cancer-associated metabolic enzyme, is commonly overexpressed in var...

journal_title：Molecular therapy. Nucleic acids

authors： Bach DH,Kim D,Bae SY,Kim WK,Hong JY,Lee HJ,Rajasekaran N,Kwon S,Fan Y,Luu TT,Shin YK,Lee J,Lee SK

更新日期：2018-06-01 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：:Transactivation of inflammatory and immune mediators in asthma is tightly regulated by nuclear factor κB (NF-κB) and signal transducer and activator of transcription 6 (STAT6). Therefore, we investigated the efficacy of simultaneous inhibition of NF-κB and STAT6 using a chimeric decoy strategy to prevent asthma exacer...

journal_title：Molecular therapy. Nucleic acids

authors： Miyake T,Miyake T,Sakaguchi M,Nankai H,Nakazawa T,Morishita R

更新日期：2018-03-02 00:00:00

abstract：:Non-coding uridine-rich small nuclear RNAs (UsnRNAs) have emerged in recent years as effective tools for exon skipping for the treatment of Duchenne muscular dystrophy (DMD), a degenerative muscular genetic disorder. We recently showed the high capacity of a recombinant adeno-associated virus (rAAV)-U7snRNA vector to ...

journal_title：Molecular therapy. Nucleic acids

authors： Domenger C,Allais M,François V,Léger A,Lecomte E,Montus M,Servais L,Voit T,Moullier P,Audic Y,Le Guiner C

更新日期：2018-03-02 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：:Recent studies have shown the effect of microRNAs on HSC activation and transformation, which is essential for the pathogenesis of liver fibrosis. In our study, we explored the role of miR-185 in liver fibrosis. Plasma miR-185 was detected in hepatitis B virus-related liver fibrosis patients (S2/3, n = 10) by Illumina...

journal_title：Molecular therapy. Nucleic acids

authors： Zhou L,Liu S,Han M,Ma Y,Feng S,Zhao J,Lu H,Yuan X,Cheng J

更新日期：2018-03-02 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：:In this study, chemical modification of 2'-deoxyinosine (2'-dI) and D-/L-isothymidine (D-/L-isoT) was performed on AS1411. They could promote the nucleotide-protein interaction by changing the local conformation. Twenty modified sequences were obtained, FCL-I and FCL-II showed the most noticeable activity improvement....

journal_title：Molecular therapy. Nucleic acids

authors： Fan X,Sun L,Li K,Yang X,Cai B,Zhang Y,Zhu Y,Ma Y,Guan Z,Wu Y,Zhang L,Yang Z

更新日期：2017-12-15 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

abstract：:Clec4a has been reported to be an immune suppressor of dendritic cells (DCs), but its potential role in cancer therapy remains to be elucidated. The present study investigated whether downregulating the expression of Clec4a via skin delivery of small hairpin RNA (shRNA) using a gene gun produced stronger host immunity...

journal_title：Molecular therapy. Nucleic acids

authors： Weng TY,Li CJ,Li CY,Hung YH,Yen MC,Chang YW,Chen YH,Chen YL,Hsu HP,Chang JY,Lai MD

更新日期：2017-12-15 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：: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：:DNA vaccine can be modified to increase protein production and modulate immune response. To enhance the efficiency of a P815 mastocytoma DNA vaccine, the P1A gene sequence was optimized by substituting specific codons with synonymous ones while modulating the number of CpG motifs. The P815A murine antigen production w...

journal_title：Molecular therapy. Nucleic acids

authors： Lopes A,Vanvarenberg K,Préat V,Vandermeulen G

更新日期：2017-09-15 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：:Antiviral strategies targeting hijacked cellular processes are less easily evaded by the virus than viral targets. If selective for viral functions, they can have a high therapeutic index. We used RNA trans-splicing to deliver the herpes simplex virus thymidine kinase-ganciclovir (HSV-tk/GCV) cell suicide system into ...

journal_title：Molecular therapy. Nucleic acids

authors： Ingemarsdotter CK,Poddar S,Mercier S,Patzel V,Lever AML

更新日期：2017-06-16 00:00:00

abstract：:Lipid nanoparticles (LNPs) have been used to successfully deliver small interfering RNAs (siRNAs) to target cells in both preclinical and clinical studies and currently are the leading systems for in vivo delivery. Here, we propose the use of an ordinary differential equation (ODE)-based model as a tool for optimizing...

journal_title：Molecular therapy. Nucleic acids

authors： Mihaila R,Ruhela D,Keough E,Cherkaev E,Chang S,Galinski B,Bartz R,Brown D,Howell B,Cunningham JJ

更新日期：2017-06-16 00:00:00

abstract：:Limited duration of transgene expression, insertional mutagenesis, and size limitations for transgene cassettes pose challenges and risk factors for many gene therapy vectors. Here, we report on physiological expression of liver phenylalanine hydroxylase (PAH) by delivery of naked DNA/minicircle (MC)-based vectors for...

journal_title：Molecular therapy. Nucleic acids

authors： Grisch-Chan HM,Schlegel A,Scherer T,Allegri G,Heidelberger R,Tsikrika P,Schmeer M,Schleef M,Harding CO,Häberle J,Thöny B

更新日期：2017-06-16 00:00:00

abstract：:Acid fibroblast growth factor (aFGF) has shown neuroprotection in Alzheimer's disease (AD) models in previous studies, yet its mechanism is still uncertain. Here we report that the efficacy of Tat-haFGF14-154 is markedly increased when loaded cationic liposomes for intranasal delivery are intranasally administered to ...

journal_title：Molecular therapy. Nucleic acids

authors： Meng T,Cao Q,Lei P,Bush AI,Xiang Q,Su Z,He X,Rogers JT,Chiu IM,Zhang Q,Huang Y

更新日期：2017-06-16 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：: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：: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：:E-selectin is an adhesion molecule expressed on the luminal surface of inflamed blood vessels that mediates hematogenous metastasis by assisting shear-resistant adhesion of circulating tumor cells to the vessel surface under dynamic blood flow. Previously, we developed an E-selectin antagonistic thioaptamer (ESTA) for...

journal_title：Molecular therapy. Nucleic acids

authors： Morita Y,Kamal M,Kang SA,Zhang R,Lokesh GL,Thiviyanathan V,Hasan N,Woo S,Zhao D,Leslie M,Suh S,Razaq W,Rui H,Gorenstein DG,Volk DE,Tanaka T

更新日期：2016-12-13 00:00:00