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                             119 results found
no title author magazine year volume issue page(s) type
1 A Biofunctional Molecular Beacon for Detecting Single Base Mutations in Cancer Cells Dong, Haiyan

5 C p.
article
2 A Broad Range of Dose Optima Achieve High-level, Long-term Gene Expression After Hydrodynamic Delivery of Sleeping Beauty Transposons Using Hyperactive SB100x Transposase Podetz-Pedersen, Kelly M

5 C p.
article
3 Acid-Sensitive Sheddable PEGylated PLGA Nanoparticles Increase the Delivery of TNF-α siRNA in Chronic Inflammation Sites Aldayel, Abdulaziz M

5 C p.
article
4 Activation of Fetal γ-globin Gene Expression via Direct Protein Delivery of Synthetic Zinc-finger DNA-Binding Domains Hossain, Mir A

5 C p.
article
5 A Gene Gun-mediated Nonviral RNA trans-splicing Strategy for Col7a1 Repair Peking, Patricia

5 C p.
article
6 A Glu-urea-Lys Ligand-conjugated Lipid Nanoparticle/siRNA System Inhibits Androgen Receptor Expression In Vivo Lee, Justin B

5 C p.
article
7 A High-Capacity Adenoviral Hybrid Vector System Utilizing the Hyperactive Sleeping Beauty Transposase SB100X for Enhanced Integration Boehme, Philip

5 C p.
article
8 Altered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy Catapano, Francesco

5 C p.
article
9 A MicroRNA Panel in Pancreatic Cyst Fluid for the Risk Stratification of Pancreatic Cysts in a Prospective Cohort Utomo, WK

5 C p.
article
10 An Amphiphilic Peptide Induces Apoptosis Through the miR29b-p53 Pathway in Cancer Cells Kim, Soyoung

5 C p.
article
11 An Efficient Method for Generation of Transgenic Rats Avoiding Embryo Manipulation Pradhan, Bhola Shankar

5 C p.
article
12 A New Transferrin Receptor Aptamer Inhibits New World Hemorrhagic Fever Mammarenavirus Entry Maier, Keith E

5 C p.
article
13 An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants Balestra, Dario

5 C p.
article
14 A Novel PD-L1-targeting Antagonistic DNA Aptamer With Antitumor Effects Lai, Wei-Yun

5 C p.
article
15 A Novel p19 Fusion Protein as a Delivery Agent for Short-interfering RNAs Danielson, Dana C

5 C p.
article
16 Antisense Oligonucleotide-based Splice Correction for USH2A-associated Retinal Degeneration Caused by a Frequent Deep-intronic Mutation Slijkerman, Radulfus WN

5 C p.
article
17 Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa Bremer, Jeroen

5 C p.
article
18 Antisense Oligonucleotide Mediated Splice Correction of a Deep Intronic Mutation in OPA1 Bonifert, Tobias

5 C p.
article
19 Application of Peptide Nucleic Acid-based Assays Toward Detection of Somatic Mosaicism Hong, Christopher S

5 C p.
article
20 Aptamer-miRNA-212 Conjugate Sensitizes NSCLC Cells to TRAIL Iaboni, Margherita

5 C p.
article
21 A Small Indel Mutant Mouse Model of Epidermolytic Palmoplantar Keratoderma and Its Application to Mutant-specific shRNA Therapy Lyu, Ya-Su

5 C p.
article
22 A Tat-conjugated Peptide Nucleic Acid Tat-PNA-DR Inhibits Hepatitis B Virus Replication In Vitro and In Vivo by Targeting LTR Direct Repeats of HBV RNA Zeng, Zhengyang

5 C p.
article
23 A Traceless Selection: Counter-selection System That Allows Efficient Generation of Transposon and CRISPR-modified T-cell Products Mezzadra, Riccardo

5 C p.
article
24 A Tumor-specific MicroRNA Recognition System Facilitates the Accurate Targeting to Tumor Cells by Magnetic Nanoparticles Yu, Yingting

5 C p.
article
25 Biodistribution and Pharmacokinetics Study of siRNA-loaded Anti-NTSR1-mAb-functionalized Novel Hybrid Nanoparticles in a Metastatic Orthotopic Murine Lung Cancer Model Perepelyuk, Maryna

5 C p.
article
26 Carrier-free Gene Silencing by Amphiphilic Nucleic Acid Conjugates in Differentiated Intestinal Cells Moroz, Elena

5 C p.
article
27 Characterization of MNK1b DNA Aptamers That Inhibit Proliferation in MDA-MB231 Breast Cancer Cells García-Recio, Eva M

5 C p.
article
28 CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector Murlidharan, Giridhar

5 C p.
article
29 Combination of microRNA-21 and microRNA-146a Attenuates Cardiac Dysfunction and Apoptosis During Acute Myocardial Infarction in Mice Huang, Wei

5 C p.
article
30 Conjugate-SELEX: A High-throughput Screening of Thioaptamer-liposomal Nanoparticle Conjugates for Targeted Intracellular Delivery of Anticancer Drugs Mu, Qingshan

5 C p.
article
31 Corrigendum to “Antisense Oligonucleotide Mediated Splice Correction of a Deep Intronic Mutation in OPA1”
5 C p.
article
32 Corrigendum to “Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method”
5 C p.
article
33 Corrigendum to “From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides”
5 C p.
article
34 Corrigendum to GFP to BFP Conversion: A Versatile Assay for the Quantification of CRISPR/Cas9-mediated Genome Editing Glaser, Astrid

5 C p.
article
35 CRISPR/Cas9-AAV Mediated Knock-in at NRL Locus in Human Embryonic Stem Cells Ge, Xianglian

5 C p.
article
36 CRISPR-Cas9 for in vivo Gene Therapy: Promise and Hurdles Dai, Wei-Jing

5 C p.
article
37 CRISPR-Cas9 System as a Versatile Tool for Genome Engineering in Human Cells Wang, Xuelian

5 C p.
article
38 Cytosolic DNA Sensor Upregulation Accompanies DNA Electrotransfer in B16.F10 Melanoma Cells Znidar, Katarina

5 C p.
article
39 Deciphering relationship between microhomology and in-frame mutation occurrence in human CRISPR-based gene knockout Chuai, Guohui

5 C p.
article
40 Defibrotide (Defitelio): A New Addition to the Stockpile of Food and Drug Administration-approved Oligonucleotide Drugs Stein, Cy

5 C p.
article
41 Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease Miniarikova, Jana

5 C p.
article
42 Design of Effective Primary MicroRNA Mimics With Different Basal Stem Conformations van den Berg, Fiona T

5 C p.
article
43 Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach Spanevello, Francesca

5 C p.
article
44 Disposition and Pharmacology of a GalNAc3-conjugated ASO Targeting Human Lipoprotein (a) in Mice Yu, Rosie Z

5 C p.
article
45 DNA as Tunable Adaptor for siRNA Polyplex Stabilization and Functionalization Heissig, Philipp

5 C p.
article
46 DNA Minicircle Technology Improves Purity of Adeno-associated Viral Vector Preparations Schnödt, Maria

5 C p.
article
47 Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain Nikan, Mehran

5 C p.
article
48 Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations Ferri, Lorenzo

5 C p.
article
49 Ecdysone Receptor-based Singular Gene Switches for Regulated Transgene Expression in Cells and Adult Rodent Tissues Lee, Seoghyun

5 C p.
article
50 Effective Prevention of Liver Fibrosis by Liver-targeted Hydrodynamic Gene Delivery of Matrix Metalloproteinase-13 in a Rat Liver Fibrosis Model Abe, Hiroyuki

5 C p.
article
51 Effects of Fibrotic Tissue on Liver-targeted Hydrodynamic Gene Delivery Kobayashi, Yuji

5 C p.
article
52 Efficient Generation of Orthologous Point Mutations in Pigs via CRISPR-assisted ssODN-mediated Homology-directed Repair Wang, Kankan

5 C p.
article
53 Efficient Modification of the CCR5 Locus in Primary Human T Cells With megaTAL Nuclease Establishes HIV-1 Resistance Romano Ibarra, Guillermo S

5 C p.
article
54 Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method Iyombe-Engembe, Jean-Paul

5 C p.
article
55 Electrotransfer parameters as a tool for controlled and targeted gene expression in skin Kos, Spela

5 C p.
article
56 Elucidation of the Biotransformation Pathways of a Galnac3-conjugated Antisense Oligonucleotide in Rats and Monkeys Shemesh, Colby S

5 C p.
article
57 Endocytosis and Endosomal Trafficking of DNA After Gene Electrotransfer In Vitro Rosazza, Christelle

5 C p.
article
58 Enhanced Anticancer Activity of PF-04691502, a Dual PI3K/mTOR Inhibitor, in Combination With VEGF siRNA Against Non–small-cell Lung Cancer Espana-Serrano, Laura

5 C p.
article
59 Episomal Nonviral Gene Therapy Vectors Slow Progression of Atherosclerosis in a Model of Familial Hypercholesterolemia Kerr, Alastair G

5 C p.
article
60 Escape Mutations, Ganciclovir Resistance, and Teratoma Formation in Human iPSCs Expressing an HSVtk Suicide Gene Kotini, Andriana G

5 C p.
article
61 E-selectin Targeting PEGylated-thioaptamer Prevents Breast Cancer Metastases Morita, Yoshihiro

5 C p.
article
62 18F-FDG PET/CT for Monitoring the Response of Breast Cancer to miR-143-Based Therapeutics by Targeting Tumor Glycolysis Miao, Ying

5 C p.
article
63 Flexible, AAV-equipped Genetic Modules for Inducible Control of Gene Expression in Mammalian Brain Dogbevia, Godwin K

5 C p.
article
64 From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides Bergsma, Atze J

5 C p.
article
65 Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice Cao, Limin

5 C p.
article
66 Full-length Dysferlin Transfer by the Hyperactive Sleeping Beauty Transposase Restores Dysferlin-deficient Muscle Escobar, Helena

5 C p.
article
67 Galaxy Workflows for Web-based Bioinformatics Analysis of Aptamer High-throughput Sequencing Data Thiel, William H

5 C p.
article
68 Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes Chamorro, Cristina

5 C p.
article
69 Gene Silencing Using 4′-thioDNA as an Artificial Template to Synthesize Short Hairpin RNA Without Inducing a Detectable Innate Immune Response Tarashima, Noriko

5 C p.
article
70 GFP to BFP Conversion: A Versatile Assay for the Quantification of CRISPR/Cas9-mediated Genome Editing Glaser, Astrid

5 C p.
article
71 Hairpin Ribozyme Genes Curtail Alcohol Drinking: from Rational Design to in vivo Effects in the Rat Sapag, Amalia

5 C p.
article
72 Hemagglutinin-targeting Artificial MicroRNAs Expressed by Adenovirus Protect Mice From Different Clades of H5N1 Infection Tang, Xinying

5 C p.
article
73 Homology Requirements for Efficient, Footprintless Gene Editing at the CFTR Locus in Human iPSCs with Helper-dependent Adenoviral Vectors Palmer, Donna J

5 C p.
article
74 Inhibition of Influenza Virus Replication by DNA Aptamers Targeting a Cellular Component of Translation Initiation Rodriguez, Paloma

5 C p.
article
75 Inhibition of miR-155 Protects Against LPS-induced Cardiac Dysfunction and Apoptosis in Mice Wang, Hui

5 C p.
article
76 In Silico Aptamer Docking Studies: From a Retrospective Validation to a Prospective Case Study'TIM3 Aptamers Binding Rabal, Obdulia

5 C p.
article
77 In vivo Editing of the Human Mutant Rhodopsin Gene by Electroporation of Plasmid-based CRISPR/Cas9 in the Mouse Retina Latella, Maria Carmela

5 C p.
article
78 In Vivo Selection Against Human Colorectal Cancer Xenografts Identifies an Aptamer That Targets RNA Helicase Protein DHX9 Mi, Jing

5 C p.
article
79 In Vivo siRNA Delivery Using JC Virus-like Particles Decreases the Expression of RANKL in Rats Hoffmann, Daniel B

5 C p.
article
80 iRNA-PseU: Identifying RNA pseudouridine sites Chen, Wei

5 C p.
article
81 Knocking Down TMPRSS2-ERG Fusion Oncogene by siRNA Could be an Alternative Treatment to Flutamide Urbinati, Giorgia

5 C p.
article
82 Lipid-based Transfection Reagents Exhibit Cryo-induced Increase in Transfection Efficiency Sork, Helena

5 C p.
article
83 Lipid Nanoparticle Delivery of siRNA to Osteocytes Leads to Effective Silencing of SOST and Inhibition of Sclerostin In Vivo Basha, Genc

5 C p.
article
84 Lipid Nanoparticle-mediated siRNA Transfer Against PCTAIRE1/PCTK1/Cdk16 Inhibits In Vivo Cancer Growth Yanagi, Teruki

5 C p.
article
85 LiPS-A3S, a human genomic site for robust expression of inserted transgenes Kotini, Andriana G

5 C p.
article
86 Long Intergenic Noncoding RNA 00511 Acts as an Oncogene in Non–small-cell Lung Cancer by Binding to EZH2 and Suppressing p57 Sun, Cheng-Cao

5 C p.
article
87 MicroRNA-27b Enhances the Hepatic Regenerative Properties of Adipose-Derived Mesenchymal Stem Cells Chen, Kuang-Den

5 C p.
article
88 microRNA-140 Inhibits Inflammation and Stimulates Chondrogenesis in a Model of Interleukin 1β-induced Osteoarthritis Karlsen, Tommy A

5 C p.
article
89 Neutrophils are Essential in Short Hairpin RNA of Indoleamine 2,3- Dioxygenase Mediated-antitumor Efficiency Liu, Kuan-Ting

5 C p.
article
90 Notch-1 Confers Chemoresistance in Lung Adenocarcinoma to Taxanes through AP-1/microRNA-451 Mediated Regulation of MDR-1 Huang, Jiayuan

5 C p.
article
91 Nucleolin-aptamer therapy in retinoblastoma: molecular changes and mass spectrometry–based imaging Subramanian, Nithya

5 C p.
article
92 Overcoming the Specific Toxicity of Large Plasmids Electrotransfer in Primary Cells In Vitro Lesueur, Léa L

5 C p.
article
93 Particle-mediated Intravenous Delivery of Antigen mRNA Results in Strong Antigen-specific T-cell Responses Despite the Induction of Type I Interferon Broos, Katrijn

5 C p.
article
94 pEVL: A Linear Plasmid for Generating mRNA IVT Templates With Extended Encoded Poly(A) Sequences Grier, Alexandra E

5 C p.
article
95 Pharmacokinetics and Pharmacodynamics of a 13-mer LNA-inhibitor-miR-221 in Mice and Non-human Primates Gallo Cantafio, Maria Eugenia

5 C p.
article
96 Pharmacokinetics and Pharmacodynamics of a 13-mer LNA-inhibitor-miR-221 in Mice and Non-human Primates
5 C p.
article
97 Poly(ester amine) Composed of Polyethylenimine and Pluronic Enhance Delivery of Antisense Oligonucleotides In Vitro and in Dystrophic mdx Mice Wang, Mingxing

5 C p.
article
98 Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases Bjurström, Carmen F

5 C p.
article
99 Reprogramming of Pancreatic Exocrine Cells AR42J Into Insulin-producing Cells Using mRNAs for Pdx1, Ngn3, and MafA Transcription Factors Koblas, Tomas

5 C p.
article
100 Reprogramming the Dynamin 2 mRNA by Spliceosome-mediated RNA Trans-splicing Trochet, Delphine

5 C p.
article
101 Restoring Ureagenesis in Hepatocytes by CRISPR/Cas9-mediated Genomic Addition to Arginase-deficient Induced Pluripotent Stem Cells Lee, Patrick C

5 C p.
article
102 RETRACTED: miR-181a Induces Macrophage Polarized to M2 Phenotype and Promotes M2 Macrophage-mediated Tumor Cell Metastasis by Targeting KLF6 and C/EBPα Bi, Jia

5 C p.
article
103 Selection and Characterization of an α6β4 Integrin blocking DNA Aptamer Berg, Katharina

5 C p.
article
104 Selection of a Novel Aptamer Against Vitronectin Using Capillary Electrophoresis and Next Generation Sequencing Stuart, Christopher H

5 C p.
article
105 Silencing CCR2 in Macrophages Alleviates Adipose Tissue Inflammation and the Associated Metabolic Syndrome in Dietary Obese Mice Kim, Jongkil

5 C p.
article
106 Silencing Myostatin Using Cholesterol-conjugated siRNAs Induces Muscle Growth Khan, Tayeba

5 C p.
article
107 Systematic Analysis of Long Noncoding RNAs in the Senescence-accelerated Mouse Prone 8 Brain Using RNA Sequencing Zhang, Shuai

5 C p.
article
108 TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs Suzuki, Shingo

5 C p.
article
109 Targeting Insulin Receptor with a Novel Internalizing Aptamer Iaboni, Margherita

5 C p.
article
110 Targeting Multidrug-resistant Staphylococci with an anti-rpoA Peptide Nucleic Acid Conjugated to the HIV-1 TAT Cell Penetrating Peptide Abushahba, Mostafa FN

5 C p.
article
111 Targeting TSLP With shRNA Alleviates Airway Inflammation and Decreases Epithelial CCL17 in a Murine Model of Asthma Chen, Yi-Lien

5 C p.
article
112 The Functionality of Minimal PiggyBac Transposons in Mammalian Cells Troyanovsky, Boris

5 C p.
article
113 The Gene Targeting Approach of Small Fragment Homologous Replacement (SFHR) Alters the Expression Patterns of DNA Repair and Cell Cycle Control Genes Pierandrei, Silvia

5 C p.
article
114 The Novel miR-9600 Suppresses Tumor Progression and Promotes Paclitaxel Sensitivity in Non–small-cell Lung Cancer Through Altering STAT3 Expression Sun, Cheng-Cao

5 C p.
article
115 The Poly-cistronic miR-23-27-24 Complexes Target Endothelial Cell Junctions: Differential Functional and Molecular Effects of miR-23a and miR-23b Li, Jia

5 C p.
article
116 Tissue-specific Calibration of Real-time PCR Facilitates Absolute Quantification of Plasmid DNA in Biodistribution Studies Ho, Joan K

5 C p.
article
117 Topical Use of Angiopoietin-like Protein 2 RNAi-loaded Lipid Nanoparticles Suppresses Corneal Neovascularization Taketani, Yukako

5 C p.
article
118 Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics Barbon, Elena

5 C p.
article
119 Validation of RNAi Silencing Efficiency Using Gene Array Data shows 18.5% Failure Rate across 429 Independent Experiments Munkácsy, Gyöngyi

5 C p.
article
                             119 results found
 
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