Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol.

abstract：:The acute hepatic porphyrias are caused by inherited enzymatic deficiencies in the heme biosynthesis pathway. Induction of the first enzyme 5-aminolevulinic acid synthase 1 (ALAS1) by triggers such as fasting or drug exposure can lead to accumulation of neurotoxic heme intermediates that cause disease symptoms. We hav...

journal_title：Molecular therapy. Nucleic acids

authors： Chan A,Liebow A,Yasuda M,Gan L,Racie T,Maier M,Kuchimanchi S,Foster D,Milstein S,Charisse K,Sehgal A,Manoharan M,Meyers R,Fitzgerald K,Simon A,Desnick RJ,Querbes W

更新日期：2015-11-03 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：: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：: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), 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：: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：:5-methylcytosine (m5C) is one of the most common and abundant post-transcriptional modifications (PTCMs) in RNA. Recent studies showed that m5C plays important roles in many biological functions such as RNA metabolism and cell fate decision. Because most experimental methods that determine m5C sites across the transcr...

journal_title：Molecular therapy. Nucleic acids

authors： Fang T,Zhang Z,Sun R,Zhu L,He J,Huang B,Xiong Y,Zhu X

更新日期：2019-12-06 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：:Deletion and mutation of the Smad4 gene are favorable events for the progression of colon cancer, which is related to the negative regulation of vascular endothelial growth factor C (VEGF-C). However, the regulatory mechanism between Smad4 and VEGF-C remains unclear. We reported first that Smad4 can increase the trans...

journal_title：Molecular therapy. Nucleic acids

authors： Li X,Lv X,Li Z,Li C,Li X,Xiao J,Liu B,Yang H,Zhang Y

更新日期：2019-12-06 00:00:00

abstract：:Precise genome editing using engineered nucleases can significantly facilitate biological studies and disease treatment. In particular, clustered regularly interspaced short palindromic repeats (CRISPR) with CRISPR-associated (Cas) proteins are a potentially powerful tool for modifying a genome by targeted cleavage of...

journal_title：Molecular therapy. Nucleic acids

authors： Cradick TJ,Qiu P,Lee CM,Fine EJ,Bao G

更新日期：2014-12-02 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：: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：: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 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：:Next-generation sequencing technology has been commonly applied to detect mitochondrial DNA (mtDNA) mutations, which are reported to be strongly associated with cancers. However, several key challenges still exist regarding bioinformatics analysis of mtDNA sequencing data that greatly affect the detection accuracy of ...

journal_title：Molecular therapy. Nucleic acids

authors： Guo S,Zhou K,Yuan Q,Su L,Liu Y,Ji X,Gu X,Guo X,Xing J

更新日期：2020-11-11 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：:MicroRNA (miR)-137 is highly expressed in the brain and plays a crucial role in the development and prognosis of glioma. In this review, we aim to summarize the latest findings regarding miR-137 in glioma cell apoptosis, proliferation, migration, invasion, angiogenesis, drug resistance, and cancer treatment. In additi...

journal_title：Molecular therapy. Nucleic acids

authors： Wang Y,Chen R,Zhou X,Guo R,Yin J,Li Y,Ma G

更新日期：2020-09-04 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

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：: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：:Currently, the most effective and durable therapeutic option for HIV-1 infection is combination antiretroviral therapy (cART). Although cART is powerful and can delay viral evolution of drug resistance for decades, it is associated with limitations, including an inability to eradicate the virus and a potential for adv...

journal_title：Molecular therapy. Nucleic acids

authors： Takahashi M,Li H,Zhou J,Chomchan P,Aishwarya V,Damha MJ,Rossi JJ

更新日期：2019-09-06 00:00:00

abstract：:Blocking the immunoinhibitory PD-1:PD-L1 pathway using monoclonal antibodies has led to dramatic clinical responses by reversing tumor immune evasion and provoking robust and durable antitumor responses. Anti-PD-1 antibodies have now been approved for the treatment of melanoma, and are being clinically tested in a num...

journal_title：Molecular therapy. Nucleic acids

authors： Prodeus A,Abdul-Wahid A,Fischer NW,Huang EH,Cydzik M,Gariépy J

更新日期：2015-04-28 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：: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：: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：: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：: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：: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：: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：: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