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Fusion of multi-source relationships and topology to infer lncRNA-protein interactions Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-04-06 Xinyu Zhang, Mingzhe Liu, Zhen Li, Linlin Zhuo, Xiangzheng Fu, Quan Zou
Long non-coding RNAs (lncRNAs) are important factors involved in biological regulatory networks. Accurately predicting lncRNA-protein interactions (LPIs) is vital for clarifying lncRNA’s functions and pathogenic mechanisms. Existing deep learning models have yet to yield satisfactory results in LPI prediction. Recently, graph autoencoders (GAEs) have seen rapid development, excelling in tasks like
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ASOptimizer: Optimizing antisense oligonucleotides through deep learning for IDO1 gene regulation Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-04-06 Gyeongjo Hwang, Mincheol Kwon, Dongjin Seo, Dae Hoon Kim, Daehwan Lee, Kiwon Lee, Eunyoung Kim, Mingeun Kang, Jin-Hyeob Ryu
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Oligonucleotide therapies for nonalcoholic steatohepatitis Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-30 Sixu Li, Feng Xiong, Songbo Zhang, Jinghua Liu, Guangping Gao, Jun Xie, Yi Wang
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Context base editing for splice correction of IVSI-110 β-thalassemia Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-30 Basma Naiisseh, Panayiota L. Papasavva, Nikoletta Y. Papaioannou, Marios Tomazou, Lola Koniali, Xenia Felekis, Constantina G. Constantinou, Maria Sitarou, Soteroula Christou, Marina Kleanthous, Carsten W. Lederer, Petros Patsali
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Identification of film-based formulations that move mRNA lipid nanoparticles out of the freezer Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-26 Trang Nguyen Kieu Doan, Madison M. Davis, Maria A. Croyle
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Inefficacy of anti-VEGF therapy reflected in VEGF-mediated photoreceptor degeneration Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-25 Xin Xu, Ni Han, Fangkun Zhao, Ruoyue Fan, Qingguo Guo, Xuefei Han, Ying Liu, Guangzuo Luo
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Optimized allele-specific silencing of the dominant-negative COL6A1 G293R substitution causing collagen VI-related dystrophy Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-22 Astrid Brull, Apurva Sarathy, Véronique Bolduc, Grace S. Chen, Riley M. McCarty, Carsten G. Bönnemann
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Enigmatic exosomal connection in lung cancer drug resistance Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-22 Sambit K. Patra, Rajeev K. Sahoo, Stuti Biswal, Shikshya S. Panda, Bijesh Kumar Biswal
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Physiologically based modeling of LNP-mediated delivery of mRNA in the vascular system Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-18 Hamideh Parhiz, Vladimir V. Shuvaev, Qin Li, Tyler E. Papp, Awurama A. Akyianu, Ruiqi Shi, Amir Yadegari, Hamna Shahnawaz, Sean C. Semple, Barbara L. Mui, Drew Weissman, Vladimir R. Muzykantov, Patrick M. Glassman
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Favorable efficacy and reduced acute neurotoxicity by antisense oligonucleotides with 2′,4′-BNA/LNA with 9-(aminoethoxy)phenoxazine Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-18 Taiki Matsubayashi, Kotaro Yoshioka, Su Su Lei Mon, Maho Katsuyama, Chunyan Jia, Takao Yamaguchi, Rintaro Iwata Hara, Tetsuya Nagata, Osamu Nakagawa, Satoshi Obika, Takanori Yokota
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Inhibition of miR-25 ameliorates cardiac and skeletal muscle dysfunction in aged mdx/utrn haploinsufficient (+/−) mice Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-17 Sacha V. Kepreotis, Jae Gyun Oh, Mina Park, Jimeen Yoo, Cholong Lee, Mark Mercola, Roger J. Hajjar, Dongtak Jeong
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Control of endothelial cell function and arteriogenesis by MEG3:EZH2 epigenetic regulation of integrin expression Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-17 Hywel Dunn-Davies, Tatiana Dudnakova, Antonella Nogara, Julie Rodor, Anita C. Thomas, Elisa Parish, Philippe Gautier, Alison Meynert, Igor Ulitsky, Paolo Madeddu, Andrea Caporali, Andrew Baker, David Tollervey, Tijana Mitić
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Accessory oligos for neuronal delivery of therapeutic siRNAs for ALS Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-13 John J. Rossi
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Temporal restriction of Cas9 expression improves CRISPR-mediated deletion efficacy and fidelity Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-11 Jesse A. Weber, Jonathan F. Lang, Ellie M. Carrell, Mohamad-Gabriel Alameh, Beverly L. Davidson
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Secondary follicles enable efficient germline mtDNA base editing at hard-to-edit site Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-11 Qin Xie, Haibo Wu, Hui Long, Caiwen Xiao, Jiaxin Qiu, Weina Yu, Xueyi Jiang, Junbo Liu, Shuo Zhang, Qifeng Lyu, Lun Suo, Yanping Kuang
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The mRNACalc webserver accounts for the N1-methylpseudouridine hypochromicity to enable precise nucleoside-modified mRNA quantification Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-11 Esteban Finol, Sarah E. Krul, Sean J. Hoehn, Xudong Lyu, Carlos E. Crespo-Hernández
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Correction of human nonsense mutation via adenine base editing for Duchenne muscular dystrophy treatment in mouse Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-06 Ming Jin, Jiajia Lin, Haisen Li, Zhifang Li, Dong Yang, Yin Wang, Yuyang Yu, Zhurui Shao, Long Chen, Zhiqiang Wang, Yu Zhang, Xiumei Zhang, Ning Wang, Chunlong Xu, Hui Yang, Wan-Jin Chen, Guoling Li
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TGF-β1-triggered BMI1 and SMAD2 cooperatively regulate miR-191 to modulate bone formation Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-06 Xiao-Fei Zhang, Zi-Xuan Wang, Bo-Wen Zhang, Kun-Peng Huang, Tian-Xing Ren, Ting Wang, Xing Cheng, Ping Hu, Wei-Hua Xu, Jin Li, Jin-Xiang Zhang, Hui Wang
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Anti-miR-141-3p maintains homeostasis between autophagy and apoptosis by targeting Yy1 in the fetal lumbosacral defecation center of rats Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-06 Yue Li, Peiqi Liu, Yifan Yao, Weilin Wang, Huimin Jia, Yuzuo Bai, Zhengwei Yuan, Zhonghua Yang
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Trans-amplifying RNA expressing functional miRNA mediates target gene suppression and simultaneous transgene expression Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-03-05 Ayşegül Yıldız, Aida Hasani, Tina Hempel, Nina Köhl, Aline Beicht, René Becker, Stefanie Hubich-Rau, Martin Suchan, Marco A. Poleganov, Ugur Sahin, Tim Beissert
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Deciphering the shield: Lnc-CCNH-8’s pivotal role in crafting PD-L1-mediated immune camouflage in hepatocellular carcinoma Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-29 Si-Yuan Song, Yamei Li, Yi Wang
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Modifying miRs for effective reprogramming of fibroblasts to cardiomyocytes Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-28 Xinghua Wang, Syeda S. Baksh, Richard E. Pratt, Victor J. Dzau, Conrad P. Hodgkinson
Reprogramming scar fibroblasts into cardiomyocytes has been proposed to reverse the damage associated with myocardial infarction. However, the limited improvement in cardiac function calls for enhanced strategies. We reported enhanced efficacy of our miR reprogramming cocktail miR combo (miR-1, miR-133a, miR-208a, and miR-499) via RNA-sensing receptor stimulation. We hypothesized that we could combine
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Exploring pulmoprotection in COVID-19: Moving toward microRNA-based theranostics Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-28 Miron Sopic
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Epigenetic and nanotechnology alliance to fight stroke-induced brain damage Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-28 Giacinto Bagetta, Maria Tiziana Corasaniti, Damiana Scuteri
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Therapeutic potential for mRNA-based IGF-I regenerative therapy Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-22 Cynthia D. Anderson
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Immunostimulatory short non-coding RNAs in the circulation of patients with tuberculosis infection Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-22 Justin Gumas, Takuya Kawamura, Megumi Shigematsu, Yohei Kirino
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Protection from cisplatin-induced hearing loss with lentiviral vector-mediated ectopic expression of the anti-apoptotic protein BCL-XL Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-19 Larissa Nassauer, Hinrich Staecker, Peixin Huang, Bryan Renslo, Madeleine Goblet, Jennifer Harre, Athanasia Warnecke, Juliane W. Schott, Michael Morgan, Melanie Galla, Axel Schambach
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Polymeric nanoparticle-based mRNA vaccine is protective against influenza virus infection in ferrets Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-19 Gijs Hardenberg, Chantal Brouwer, Rachelle van Gemerden, Nicola J. Jones, Anthony C. Marriott, Jaap Rip
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Increasing angiogenesis factors in hypoxic diabetic wounds using siRNA nanotherapeutics Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-19 Adam Astrada
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Single-cell transcriptome profiling implicates the psychological stress-induced disruption of spermatogenesis Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-19 Rufeng Li, Yuefeng Du, Kang Li, Xiaofan Xiong, Lingyu Zhang, Chen Guo, Shanfeng Gao, Yufei Yao, Yungang Xu, Juan Yang
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Metabolism pathway-based subtyping in endometrial cancer: An integrated study by multi-omics analysis and machine learning algorithms Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-16 Xiaodie Liu, Wenhui Wang, Xiaolei Zhang, Jing Liang, Dingqing Feng, Yuebo Li, Ming Xue, Bin Ling
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LSpECifying transgene expression Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-16 Andrea Annoni, Alessio Cantore
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STAT6-targeting antisense oligonucleotides against solitary fibrous tumor Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-15 Yi Li, Jose L. Mondaza-Hernandez, David S. Moura, Alexey S. Revenko, Angelica Tolentino, John T. Nguyen, Nam Tran, Clark A. Meyer, Jose Merino-Garcia, Rafael Ramos, Davide Di Lernia, Javier Martin-Broto, Heather N. Hayenga, Leonidas Bleris
Solitary fibrous tumor (SFT) is a rare, non-hereditary soft tissue sarcoma thought to originate from fibroblastic mesenchymal stem cells. The etiology of SFT is thought to be due to an environmental intrachromosomal gene fusion between NGFI-A-binding protein 2 (NAB2) and signal transducer and activator protein 6 (STAT6) genes on chromosome 12, wherein the activation domain of STAT6 is fused with the
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Therapeutic role of miR-19a/b protection from influenza virus infection in patients with coronary heart disease Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-15 Yanan Xing, Lin Chen, Bin Hu, Yi Li, Huan Mai, Gaojian Li, Shuyi Han, Ye Wang, Yanyi Huang, Ying Tian, Wei Zhang, Yan Gao, Hongxuan He
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Intrathecal administration of a novel siRNA modality extends survival and improves motor function in the SOD1G93A ALS mouse model Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-15 Chunling Duan, Moorim Kang, Xiaojie Pan, Zubao Gan, Vera Huang, Guanlin Li, Robert F. Place, Long-Cheng Li
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Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4 Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-13 Hali Sai, Bethany Ollington, Farah O. Rezek, Niuzheng Chai, Amelia Lane, Anastasios Georgiadis, James Bainbridge, Michel Michaelides, Almudena Sacristan-Reviriego, Pedro R.L. Perdigão, Amy Leung, Jacqueline van der Spuy
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Unique quinoline orientations shape the modified aptamer to sclerostin for enhanced binding affinity and bone anabolic potential Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-13 Amu Gubu, Yuan Ma, Sifan Yu, Huarui Zhang, Zefeng Chen, Shuaijian Ni, Razack Abdullah, Huan Xiao, Yihao Zhang, Hong Dai, Hang Luo, Yuanyuan Yu, Luyao Wang, Hewen Jiang, Ning Zhang, Yuan Tian, Haitian Li, Aiping Lu, Baoting Zhang, Ge Zhang
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Synthetic mRNA delivered to human cells leads to expression of Cpl-1 bacteriophage-endolysin with activity against Streptococcus pneumoniae Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-08 Moritz K. Jansson, Dat Tien Nguyen, Stefan Mikkat, Carolin Warnke, Marc Benjamin Janssen, Philipp Warnke, Bernd Kreikemeyer, Nadja Patenge
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MiR-128-3p – a gray eminence of the human central nervous system Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-06 Klaudia Kiel, Sylwia Katarzyna Król, Agnieszka Bronisz, Jakub Godlewski
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MiR-449a antagonizes EMT through IL-6-mediated trans-signaling in laryngeal squamous cancer Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-06 Alessia Maria Cossu, Federica Melisi, Teresa Maria Rosaria Noviello, Lucia Stefania Pasquale, Piera Grisolia, Carla Reale, Marco Bocchetti, Michela Falco, Chiara Tammaro, Nunzio Accardo, Francesco Longo, Salvatore Allosso, Massimo Mesolella, Raffaele Addeo, Francesco Perri, Alessandro Ottaiano, Filippo Ricciardiello, Evzen Amler, Concetta Ambrosino, Gabriella Misso, Michele Ceccarelli, Michele Caraglia
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Recent advances in CRISPR-Cas9-based genome insertion technologies Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-05 Xinwen Chen, Jingjing Du, Shaowei Yun, Chaoyou Xue, Yao Yao, Shuquan Rao
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MHGTMDA: Molecular heterogeneous graph transformer based on biological entity graph for miRNA-disease associations prediction Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-05 Haitao Zou, Boya Ji, Meng Zhang, Fen Liu, Xiaolan Xie, Shaoliang Peng
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Oligo-PROTAC strategy for cell-selective and targeted degradation of activated STAT3 Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-05 Jeremy Hall, Zhuoran Zhang, Supriyo Bhattacharya, Dongfang Wang, Marice Alcantara, Yong Liang, Piotr Swiderski, Stephen Forman, Larry Kwak, Nagarajan Vaidehi, Marcin Kortylewski
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Anti-miRNA103/107 encapsulated in transferrin-conjugated lipid nanoparticles crosses blood-brain barrier and reduces brain ischemic damage Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-02 Pasquale Cepparulo, Ornella Cuomo, Virginia Campani, Antonio Vinciguerra, Maria Josè Sisalli, Valeria Nele, Serenella Anzilotti, Valeria Valsecchi, Antonella Casamassa, Paola Brancaccio, Antonella Scorziello, Giuseppe De Rosa, Lucio Annunziato, Giuseppe Pignataro
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mitoTALEN reduces the mutant mtDNA load in neurons Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-02 Sandra R. Bacman, Jose Domingo Barrera-Paez, Milena Pinto, Derek Van Booven, James B. Stewart, Anthony J. Griswold, Carlos T. Moraes
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Quantitative proteomics of the miR-301a/SOCS3/STAT3 axis reveals underlying autism and anxiety-like behavior Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-02 Xun Li, Qi Fu, Mingtian Zhong, Yihao Long, Fengyun Zhao, Yanni Huang, Zizhu Zhang, Min Wen, Kaizhao Chen, Rongqing Chen, Xiaodong Ma
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Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-02 Sriram Vaidyanathan, Jenny L. Kerschner, Alekh Paranjapye, Vrishti Sinha, Brian Lin, Tracy A. Bedrosian, Adrian J. Thrasher, Giandomenico Turchiano, Ann Harris, Matthew H. Porteus
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Tissue pharmacokinetics of antisense oligonucleotides Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-02 Erica Bäckström, Alessandro Bonetti, Per Johnsson, Stefan Öhlin, Anders Dahlén, Patrik Andersson, Shalini Andersson, Peter Gennemark
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Hair cell-specific Myo15 promoter-mediated gene therapy rescues hearing in DFNB9 mouse model Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-02-02 Hui Wang, MengZhao Xun, Honghai Tang, Jingjing Zhao, Shaowei Hu, Longlong Zhang, Jun Lv, Daqi Wang, Yuxin Chen, Jianping Liu, Geng-lin Li, Wuqing Wang, Yilai Shu, Huawei Li
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A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-30 Nina Schneider, Ricky Steinberg, Amit Ben-David, Johanna Valensi, Galit David-Kadoch, Zohar Rosenwasser, Eyal Banin, Erez Y. Levanon, Dror Sharon, Shay Ben-Aroya
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Engineering cGAS-agonistic oligonucleotides as therapeutics for cancer immunotherapy Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-24 Shurong Zhou, Ting Su, Furong Cheng, Janet Cole, Xiang Liu, Bei Zhang, Shaheer Alam, Jinze Liu, Guizhi Zhu
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Global characterization of RNA editing in genetic regulation of multiple ovarian cancer subtypes Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-24 Yulan Wang, Jing Wu, Jian Zhao, Tianyi Xu, Meng Zhang, Jingjing Liu, Yixuan Wang, Quan Wang, Xiaofeng Song
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Exosomal noncoding RNA: A potential therapy for retinal vascular diseases Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-24 Jong-Ik Heo, Juhee Ryu
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Functional requirement of alternative splicing in epithelial-mesenchymal transition of pancreatic circulating tumor Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-24 Lixing Xiao, Haoxiu Sun, Rui Cheng, Rongrong Yang, Xiyun Jin, Zhaochun Xu, Yideng Cai, Yuexin Yang, Fenglan Pang, Guangfu Xue, Pingping Wang, Qinghua Jiang, Huan Nie
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Preclinical evaluation of CRISPR-based therapies for Noonan syndrome caused by deep-intronic LZTR1 variants Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-23 Carolin Knauer, Henrike Haltern, Eric Schoger, Sebastian Kügler, Lennart Roos, Laura C. Zelarayán, Gerd Hasenfuss, Wolfram-Hubertus Zimmermann, Bernd Wollnik, Lukas Cyganek
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Lnc-CCNH-8 promotes immune escape by up-regulating PD-L1 in hepatocellular carcinoma Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-23 Bixing Zhao, Xiaoyuan Zheng, Yang Wang, Niangmei Cheng, Yue Zhong, Yang Zhou, Jingyun Huang, Fei Wang, Xin Qi, Qiuyu Zhuang, Yingchao Wang, Xiaolong Liu
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SpCas9-HF1 enhances accuracy of cell cycle-dependent genome editing by increasing HDR efficiency, and by reducing off-target effects and indel rates Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-23 Daisuke Matsumoto, Erina Matsugi, Kanae Kishi, Yuto Inoue, Kiyomi Nigorikawa, Wataru Nomura
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Sharper scissors: How TREX2 fine-tunes CRISPR-Cas9 nickases for precise editing Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-19 Marc Güell
Abstract not available
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Promising strategies employing nucleic acids as antimicrobial drugs Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-18 Luís Moreira, Nuno M. Guimarães, Rita S. Santos, Joana A. Loureiro, Maria C. Pereira, Nuno F. Azevedo
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Topical tissue nanotransfection of Prox1 is effective in the prophylactic management of lymphedema Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-18 Ganesh Mohan, Imran Khan, Colby R. Neumann, Miguel D. Jorge, Shahnur Ahmed, Luci Hulsman, Mithun Sinha, Gayle M. Gordillo, Chandan K. Sen, Aladdin H. Hassanein