Development of a Facile Approach for Generating Chemically Modified CRISPR/Cas9 RNA.

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：:Mitochondrial DNA (mtDNA) haplogroups are associated with various types of cancer; however, the molecular mechanisms by which mtDNA haplogroups affect primary hepatocellular carcinoma (HCC) are not known. In this study, we carried out a case-control study on 388 HCC patients and 511 geographically matched asymptomatic...

journal_title：Molecular therapy. Nucleic acids

authors： Hua S,Li M,Zhao Q,Wang J,Zhou Y,Liu J,Fang H,Jiang M,Shen L

更新日期：2019-12-06 00:00:00

abstract：:Targeted transcriptional modulation in the central nervous system (CNS) can be achieved by adeno-associated virus (AAV) delivery of CRISPR activation (CRISPRa) and interference (CRISPRi) transgenes. To enable AAV packaging, we constructed minimal CRISPRa and CRISPRi transgenes by fusing catalytically inactive Staphylo...

journal_title：Molecular therapy. Nucleic acids

authors： Lau CH,Ho JW,Lo PK,Tin C

更新日期：2019-06-07 00:00:00

abstract：:Menin displays either tumor suppression or promotion functions in a context-dependent manner. Previously, we proposed that Menin acts as a tumor suppressor by inhibiting cell growth in pancreatic ductal adenocarcinoma (PDAC), whereas the relationship between the Menin expression and overall survival rate of PDAC patie...

journal_title：Molecular therapy. Nucleic acids

authors： Cheng P,Chen Y,He TL,Wang C,Guo SW,Hu H,Ni CM,Jin G,Zhang YJ

更新日期：2019-12-06 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：: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：: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：: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：: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：: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：: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：: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：: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：: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：:Exosomes are membrane-enclosed nanovesicles that shuttle active cargoes, such as mRNAs and microRNAs (miRNAs), between different cells. Mesenchymal stem cells (MSCs) are able to migrate to the tumor sites and exert complex functions over tumor progress. We investigated the effect of human bone marrow-derived MSC (BMSC...

journal_title：Molecular therapy. Nucleic acids

authors： Che Y,Shi X,Shi Y,Jiang X,Ai Q,Shi Y,Gong F,Jiang W

更新日期：2019-12-06 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：: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：: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：: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：: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：:A hexanucleotide GGGGCC expansion in intron 1 of chromosome 9 open reading frame 72 (C9orf72) gene is the most frequent cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The corresponding repeat-containing sense and antisense transcripts cause a gain of toxicity through the accumulation o...

journal_title：Molecular therapy. Nucleic acids

authors： Martier R,Liefhebber JM,García-Osta A,Miniarikova J,Cuadrado-Tejedor M,Espelosin M,Ursua S,Petry H,van Deventer SJ,Evers MM,Konstantinova P

更新日期：2019-06-07 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：: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：: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：: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：: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：: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：:Hepatocellular carcinoma (HCC) is one of the most lethal human malignancies. Chemotherapeutic agents, such as sorafenib and lenvatinib, can improve the outcomes of HCC patients. Nevertheless, chemoresistance has become a major hurdle in the effective treatment of HCC. Noncoding RNAs (ncRNAs), including mircoRNAs (miRN...

journal_title：Molecular therapy. Nucleic acids

authors： Wang M,Yu F,Chen X,Li P,Wang K

更新日期：2020-09-04 00:00:00

abstract：:Neovascular age-related macular degeneration (AMD) is characterized by the formation of choroidal neovascularization, which is responsible for more than 80% of cases of severe vision loss. Ubiquitin protein ligase E3D (UBE3D) gene missense has been proven to be associated with neovascular AMD in the East Asian populat...

journal_title：Molecular therapy. Nucleic acids

authors： Xia H,Zhang Q,Shen Y,Bai Y,Ma X,Zhang B,Qi Y,Zhang J,Hu Q,Du W,Zhu L,Zhou P,Wang B,Xu H,Huang L,Li X

更新日期：2020-06-05 00:00:00