Size unbiased representative enzymatically generated RNAi (SURER) library and application for RNAi therapeutic screens.

abstract：:A promising strategy for increased intracellular delivery of nucleic acids with the benefit for targeting is photochemical internalization (PCI). PCI relies on the use of a photosensitizing compound that photochemically destroys membranes in the endocytic pathway after illumination, resulting in cytosolic transfer of ...

journal_title：Nucleic acid therapeutics

authors： Jørgensen JA,Longva AS,Hovig E,Bøe SL

更新日期：2013-04-01 00:00:00

abstract：:Adjuvants are important components of vaccine formulations. Effective adjuvants line innate and adaptive immunity by signaling through pathogen recognition receptors. Synthetic cytosine-phosphate-guanine (CpG) oligodeoxynucleotides (ODNs) have been shown to have potentials as adjuvants for vaccines. However, the immun...

journal_title：Nucleic acid therapeutics

authors： Chu CY,Lee SC,Liu SS,Lin YM,Shen PC,Yu C,Lee KH,Zhao X,Lee JW

更新日期：2011-10-01 00:00:00

abstract：:Gold nanorods (AuNRs) were used as spectroscopic sensing elements to detect specific DNA sequences with a single-base mismatch sensitivity. The assay was based on the observation that the stabilizing repulsive forces between CTA(+)-coated AuNRs can be removed by citrate ions, which causes aggregation among AuNRs; wher...

journal_title：Nucleic acid therapeutics

authors： Kanjanawarut R,Yuan B,XiaoDi S

更新日期：2013-08-01 00:00:00

abstract：:Treating intracellular pathogens remains a considerable medical challenge because of the inefficient intracellular delivery of antimicrobials and the frequent emergence of bacterial resistance to therapeutic agents deemed the drugs of last resort. We investigated the capability of antisense peptide nucleic acids (PNAs...

journal_title：Nucleic acid therapeutics

authors： Alajlouni RA,Seleem MN

更新日期：2013-10-01 00:00:00

abstract：:The tissue half-life of second-generation antisense oligonucleotide drugs (ASOs) is generally longer than traditional small molecule therapeutics. Thus, a strategy to reverse the activity of antisense drugs is warranted in certain settings. In this study, we describe a strategy employing the administration of a comple...

journal_title：Nucleic acid therapeutics

authors： Crosby JR,Zhao C,Zhang H,MacLeod AR,Guo S,Monia BP

更新日期：2015-12-01 00:00:00

abstract：BACKGROUND:Ischemia-reperfusion (I/R) is the main cause of acute kidney injury (AKI) in patients. We investigated renal microRNA (miRNA) expression profiles and the time course of changes in selected miRNA expressions after renal I/R to characterize the miRNA network activated during development and recovery from AKI. ...

journal_title：Nucleic acid therapeutics

authors： Kaucsár T,Révész C,Godó M,Krenács T,Albert M,Szalay CI,Rosivall L,Benyó Z,Bátkai S,Thum T,Szénási G,Hamar P

更新日期：2013-10-01 00:00:00

abstract：:U1 small nuclear interference (U1i) has recently been described as a novel gene silencing mechanism. U1i employs short oligonucleotides, so-called U1 adaptors, for specific gene knockdown, expanding the field of current silencing strategies that are primarily based on RNA interference (RNAi) or antisense. Despite the ...

journal_title：Nucleic acid therapeutics

authors： Weirauch U,Grünweller A,Cuellar L,Hartmann RK,Aigner A

更新日期：2013-08-01 00:00:00

abstract：:RNA activation (RNAa) is a mechanism of gene activation triggered by promoter-targeted small double-stranded RNA (dsRNA), also known as small activating RNA (saRNA). p21(WAF1/CIP1) (p21) is a putative tumor suppressor gene due to its role as a key negative regulator of the cell cycle and cell proliferation. It is freq...

journal_title：Nucleic acid therapeutics

authors： Kosaka M,Kang MR,Yang G,Li LC

更新日期：2012-10-01 00:00:00

abstract：:Antisense oligonucleotides (ASOs) regulate gene expression by binding to complementary target RNA, and ASOs can be designed to take advantage of a growing array of post RNA binding molecular mechanisms. Intracellular trafficking of ASOs influences their efficacy. We have identified a number of membrane-less structures...

journal_title：Nucleic acid therapeutics

authors： Wang Y,Shen W,Liang XH,Crooke ST

更新日期：2019-12-01 00:00:00

abstract：:Decreased production of erythropoietin (EPO) causes anemia in patients with chronic kidney disease, and recombinant human EPO is used to treat renal failure associated anemia. The liver, the main EPO-producing organ in utero, maintains the capacity to produce EPO in the adult but in insufficient quantities to restore ...

journal_title：Nucleic acid therapeutics

authors： Abrams MT,Koser M,Burchard J,Strapps W,Mehmet H,Gindy M,Zaller D,Sepp-Lorenzino L,Stickens D

更新日期：2014-12-01 00:00:00

abstract：:Orphan drugs, including antisense oligonucleotides (AONs), siRNAs/miRNAs, Cas9 nuclease, and recombinant genes, have recently been made available for rare diseases. However, the main bottleneck for these new therapies is delivery. Drugs/synthetic genes need to reach the affected tissues with minimal off-target effects...

journal_title：Nucleic acid therapeutics

authors： Falzarano MS,Argenziano M,Marsollier AC,Mariot V,Rossi D,Selvatici R,Dumonceaux J,Cavalli R,Ferlini A

更新日期：2020-07-13 00:00:00

abstract：:Decreases in platelet (PLT) counts observed in nonhuman primates (NHPs) given 2'-O-methoxyethyl modified antisense inhibitors (2'-MOE ASOs) have been reported, but the incidence and severity of the change vary considerably between sequences, studies, and animals. This article will broadly illustrate the spectrum of ef...

journal_title：Nucleic acid therapeutics

authors： Henry SP,Narayanan P,Shen L,Bhanot S,Younis HS,Burel SA

更新日期：2017-08-01 00:00:00

abstract：:Antisense oligonucleotide (AON)-mediated exon skipping is a promising therapeutic approach for Duchenne muscular dystrophy that is currently being tested in various clinical trials. This approach is based on restoring the open reading frame of dystrophin transcripts resulting in shorter but partially functional dystro...

journal_title：Nucleic acid therapeutics

authors： Jirka SM,Heemskerk H,Tanganyika-de Winter CL,Muilwijk D,Pang KH,de Visser PC,Janson A,Karnaoukh TG,Vermue R,'t Hoen PA,van Deutekom JC,Aguilera B,Aartsma-Rus A

更新日期：2014-02-01 00:00:00

abstract：:Antisense synthetic oligonucleotides have been developed as potential gene-targeted therapeutics. We previously reported polymerase-endonuclease amplification reaction (PEAR) for amplification of natural and 5'-O-(1-thiotriphosphate) (S)-modified oligonucleotides. Here, we extended the PEAR technique for enzymatic pre...

journal_title：Nucleic acid therapeutics

authors： Wang X,Zhang J,Li Y,Chen G,Wang X

更新日期：2015-02-01 00:00:00

abstract：:Single-stranded (ss) 2'-fluoro (2'-F)-modified oligonucleotides (ONs) with a full phosphorothioate (PS) backbone have been reported to be cytotoxic and cause DNA double-strand breaks (DSBs) when transfected into HeLa cells. However, the molecular determinants of these effects have not been fully explored. In this stud...

journal_title：Nucleic acid therapeutics

authors： Janas MM,Jiang Y,Schlegel MK,Waldron S,Kuchimanchi S,Barros SA

更新日期：2017-02-01 00:00:00

abstract：:Cluster of differentiation 24 (CD24) is a cell surface glycoprotein, which is largely present on hematopoietic cells and many types of solid tumor cells. CD24 is known to be involved in a wide range of downstream signaling pathways and neural development, yet the underlying mechanisms are poorly understood. Moreover, ...

journal_title：Nucleic acid therapeutics

authors： Fafińska J,Czech A,Sitz T,Ignatova Z,Hahn U

更新日期：2018-12-01 00:00:00

abstract：:Double-stranded RNAs can target gene promoters and inhibit transcription. To date, most research has focused on synthetic RNA duplexes. Transcriptional silencing by hairpin RNAs would facilitate a better understanding of endogenous RNA-mediated regulation of transcription within cells. Here we examine transcriptional ...

journal_title：Nucleic acid therapeutics

authors： Chu Y,Kalantari R,Dodd DW,Corey DR

更新日期：2012-06-01 00:00:00

abstract：:This white paper summarizes the current consensus of the Japanese Research Working Group for the ICH S6 & Related Issues (WGS6) on strategies for the nonclinical safety assessment of oligonucleotide-based therapeutics (ONTs), specifically focused on the similarities and differences to biotechnology-derived pharmaceuti...

journal_title：Nucleic acid therapeutics

authors： Hirabayashi Y,Maki K,Kinoshita K,Nakazawa T,Obika S,Naota M,Watanabe K,Suzuki M,Arato T,Fujisaka A,Fueki O,Ito K,Onodera H

更新日期：2021-01-19 00:00:00

abstract：:C promoter binding factor 1 (CBF1) (alias RBPJ) is a critical transcription factor involved in Notch signaling. The activation of Notch signaling through CBF1 maintains the angiostatic state of endothelial cells suppressing angiogenesis, that is, the formation of new blood vessels. Vascular endothelial growth factor (...

journal_title：Nucleic acid therapeutics

authors： Tezuka-Kagajo M,Maekawa M,Ogawa A,Hatta Y,Ishii E,Eguchi M,Higashiyama S

更新日期：2020-12-01 00:00:00

abstract：:Murine Krebs-2 tumor-initiating stem cells are known to natively internalize extracellular double-stranded DNA fragments. Being internalized, these fragments interfere in the repair of chemically induced interstrand cross-links. In the current investigation, 756 bp polymerase chain reaction (PCR) product containing bu...

journal_title：Nucleic acid therapeutics

authors： Dolgova EV,Evdokimov AN,Proskurina AS,Efremov YR,Bayborodin SI,Potter EA,Popov AA,Petruseva IO,Lavrik OI,Bogachev SS

更新日期：2019-10-01 00:00:00

abstract：:Activated protein C (APC) is a serine protease with anticoagulant and cytoprotective activities. Nonanticoagulant APC mutants show beneficial effects as cytoprotective agents. To study, if such biased APC signaling can be achieved by APC-binding ligands, the aptamer technology has been used. A G-quadruplex-containing ...

journal_title：Nucleic acid therapeutics

authors： Hamedani NS,Müller J,Tolle F,Rühl H,Pezeshkpoor B,Liphardt K,Oldenburg J,Mayer G,Pötzsch B

更新日期：2020-10-01 00:00:00

abstract：:ISIS 481464 is a constrained ethyl (cEt) modified phosphorothioate antisense oligonucleotide (ASO) targeting signal transducer and activator of transcription 3 (STAT3) studied in mice and monkey to support oncology clinical trials. Six-week toxicology studies were performed in mice and cynomolgus monkey (up to 70 and ...

journal_title：Nucleic acid therapeutics

authors： Burel SA,Han SR,Lee HS,Norris DA,Lee BS,Machemer T,Park SY,Zhou T,He G,Kim Y,MacLeod AR,Monia BP,Lio S,Kim TW,Henry SP

更新日期：2013-06-01 00:00:00

abstract：:This review will cover the current strategies that are being adopted to efficiently deliver small interfering RNA using nonviral vectors, including the use of polymers such as polyethylenimine, poly(lactic-co-glycolic acid), polypeptides, chitosan, cyclodextrin, dendrimers, and polymers-containing different nanopartic...

journal_title：Nucleic acid therapeutics

authors： Singha K,Namgung R,Kim WJ

更新日期：2011-06-01 00:00:00

abstract：:DNAzymes of the 10-23 family represent an important class of antisense molecules with implications for therapeutic treatment of diseases. These molecules are single-stranded oligodeoxynucleotides combining the high specificity of oligonucleotide base pairing with an inherent RNA-cleaving enzymatic activity. However, l...

journal_title：Nucleic acid therapeutics

authors： Dicke T,Pali-Schöll I,Kaufmann A,Bauer S,Renz H,Garn H

更新日期：2012-04-01 00:00:00

abstract：:The highly conserved and ubiquitous molecular chaperone heat shock protein 70 (Hsp70) plays a critical role in protein homeostasis (proteostasis). Controlled by its ATPase activity, Hsp70 cycles between two conformations, Hsp70-ATP and Hsp70-ADP, to bind and release its substrate. Chemical tools with distinct modes of...

journal_title：Nucleic acid therapeutics

authors： Thirunavukarasu D,Shi H

更新日期：2015-04-01 00:00:00

abstract：:State-of-the-art small interfering RNA (siRNA) therapeutics such as givosiran and fitusiran are constructed from three variable components: a fully-modified RNA core that conveys metabolic stability, a targeting moiety that mediates target-cell uptake, and a linker. This structural complexity poses challenges for meta...

journal_title：Nucleic acid therapeutics

authors： Berk C,Civenni G,Wang Y,Steuer C,Catapano CV,Hall J

更新日期：2020-04-20 00:00:00

abstract：:Human telomeric DNA has the ability to fold into a 4-stranded G-quadruplex structure. Several G-quadruplex ligands are known to stabilize the structure and thereby inhibit telomerase activity. Such ligands have demonstrated efficient telomerase inhibition in dilute conditions, but under molecular crowding conditions m...

journal_title：Nucleic acid therapeutics

authors： Agarwal T,Pradhan D,Géci I,El-Madani AM,Petersen M,Pedersen EB,Maiti S

更新日期：2012-12-01 00:00:00

abstract：:Influenza A virus (IAV) affects 5%-10% of the world's population every year. Through genome changes, many IAV strains develop resistance to currently available anti-influenza therapeutics. Therefore, there is an urgent need to find new targets for therapeutics against this important human respiratory pathogen. In this...

journal_title：Nucleic acid therapeutics

authors： Lenartowicz E,Nogales A,Kierzek E,Kierzek R,Martínez-Sobrido L,Turner DH

更新日期：2016-10-01 00:00:00

abstract：:Pseudomonas aeruginosa is an opportunistic pathogen causing severe infections in hospital settings, especially with immune compromised patients, and the increasing prevalence of multidrug resistant strains urges search for new drugs with novel mechanisms of action. In this study we introduce antisense peptide-peptide ...

journal_title：Nucleic acid therapeutics

authors： Ghosal A,Nielsen PE

更新日期：2012-10-01 00:00:00

abstract：:AS1411 is a g-quadruplex-forming aptamer capable of selectively entering cancer cells by nucleolin receptor-mediated uptake. In this study, we investigated the cell internalization properties and plasticity of AS1411 carrying different locked nucleic acid-containing cargo oligonucleotides (ONs) for delivery into A549 ...

journal_title：Nucleic acid therapeutics

authors： Gissberg O,Zaghloul EM,Lundin KE,Nguyen CH,Landras-Guetta C,Wengel J,Zain R,Smith CI

更新日期：2016-06-01 00:00:00