Hairpin Ribozyme Genes Curtail Alcohol Drinking: from Rational Design to in vivo Effects in the Rat.

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：: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：: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：: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：: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

abstract：:Gene knockdown using short hairpin RNAs (shRNAs) is a promising strategy for targeting dominant mutations; however, delivering too much shRNA can disrupt the processing of endogenous microRNAs (miRNAs) and lead to toxicity. Here, we sought to understand the effect that excessive shRNAs have on muscle miRNAs by treatin...

journal_title：Molecular therapy. Nucleic acids

authors： Course MM,Gudsnuk K,Desai N,Chamberlain JR,Valdmanis PN

更新日期：2020-03-06 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：:Reactivation of γ-globin expression has been shown to ameliorate disease phenotypes associated with mutations in the adult β-globin gene, including sickle cell disease. Specific mutations in the promoter of the γ-globin genes are known to prevent repression of the genes in the adult and thus lead to hereditary persist...

journal_title：Molecular therapy. Nucleic acids

authors： Hossain MA,Shen Y,Knudson I,Thakur S,Stees JR,Qiu Y,Pace BS,Peterson KR,Bungert J

更新日期：2016-10-18 00:00:00

abstract：:Osteoarthritis (OA) is the most common degenerative joint disease. One of the main pathogenic factors of OA is thought to be inflammation. Other factors associated with OA are dysregulation of microRNAs, reduced autophagic activity, oxidative stress, and altered metabolism. microRNAs are small non-coding RNAs that are...

journal_title：Molecular therapy. Nucleic acids

authors： Al-Modawi RN,Brinchmann JE,Karlsen TA

更新日期：2019-09-06 00:00:00

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：: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：: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：: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：: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：: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：:As the world population grows, muscle atrophy leading to muscle wasting could become a bigger risk. Long noncoding RNAs (lncRNAs) are known to play important roles in muscle growth and muscle atrophy. Meanwhile, it has recently come to light that many putative small open reading frames (sORFs) are hidden in lncRNAs; h...

journal_title：Molecular therapy. Nucleic acids

authors： Cai B,Li Z,Ma M,Zhang J,Kong S,Abdalla BA,Xu H,Jebessa E,Zhang X,Lawal RA,Nie Q

更新日期：2020-12-10 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：: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：:Numerous studies have suggested that dysregulated long noncoding RNAs (lncRNAs) contributed to the development and progression of many cancers. lncRNA OIP5 antisense RNA 1 (OIP5-AS1) has been reported to be increased in several cancers. However, the roles of OIP5-AS1 in liver hepatocellular carcinoma (LIHC) remain to ...

journal_title：Molecular therapy. Nucleic acids

authors： Ma YS,Chu KJ,Ling CC,Wu TM,Zhu XC,Liu JB,Yu F,Li ZZ,Wang JH,Gao QX,Yi B,Wang HM,Gu LP,Li L,Tian LL,Shi Y,Jiang XQ,Fu D,Zhang XW

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：: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：: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：:The knockdown of myostatin, a negative regulator of skeletal muscle mass may have important implications in disease conditions accompanied by muscle mass loss like cancer, HIV/AIDS, sarcopenia, muscle atrophy, and Duchenne muscular dystrophy (DMD). In DMD patients, where major muscle loss has occurred due to a lack of...

journal_title：Molecular therapy. Nucleic acids

authors： Malerba A,Kang JK,McClorey G,Saleh AF,Popplewell L,Gait MJ,Wood MJ,Dickson G

更新日期：2012-12-18 00:00:00

abstract：:Long non-coding RNA (lncRNA) H19 is associated with inflammatory diseases, but the molecular mechanism of H19 in the inflammatory process of ankylosing spondylitis (AS) is unclear. Here, we investigated the role of H19 and its downstream molecules in the inflammation of AS by microarray analysis, qRT-PCR, western blot...

journal_title：Molecular therapy. Nucleic acids

authors： Zhang X,Ji S,Cai G,Pan Z,Han R,Yuan Y,Xu S,Yang J,Hu X,Chen M,Wu M,Ma Y,Deng J,Gao X,Guan S,Xu S,Shuai Z,Laslett L,Pan F

更新日期：2020-03-06 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：:Highly active antiretroviral therapy (HAART) has significantly improved the quality of life and the life expectancy of HIV-infected individuals. Still, drug-induced side effects and emergence of drug-resistant viral variants remain important issues that justify the exploration of alternative therapeutic options. One s...

journal_title：Molecular therapy. Nucleic acids

authors： Centlivre M,Legrand N,Klamer S,Liu YP,Jasmijn von Eije K,Bohne M,Rijnstra ES,Weijer K,Blom B,Voermans C,Spits H,Berkhout B

更新日期：2013-09-03 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：:Keratinocyte proliferation and migration are crucial steps during skin wound healing. The functional role of microRNAs (miRs) remains relatively unknown during this process. miR-93 levels have been reported to increase within 24 h of skin wound healing; however, whether miR-93-3p or miR-93-5p plays a specific role in ...

journal_title：Molecular therapy. Nucleic acids

authors： Feng X,Zhou S,Cai W,Guo J

更新日期：2020-11-26 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：: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