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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
RNA therapeutics are an emerging, powerful class of drugs with potential applications in a wide range of disorders. A central challenge in their development is the lack of clear pharmacokinetic-pharmacodynamic relationship, in part due to the significant delay between the kinetics of RNA delivery and the onset of pharmacologic response. To bridge this gap, we have developed a physiologically-based
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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
An increasing number of antisense oligonucleotides (ASOs) have been approved for clinical use. However, improvements of both efficacy and safety in the central nervous system (CNS) are crucial for the treatment with CNS diseases. We aimed to overcome the crucial issues by our development of various gapmer ASOs with a novel nucleoside derivative including a 2′,4′-BNA/LNA with 9-(aminoethoxy)phenoxazine
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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
Dystrophic cardiomyopathy is a significant feature of Duchenne Muscular Dystrophy (DMD). Increased cardiomyocyte cytosolic calcium (Ca) and interstitial fibrosis are major pathophysiological hallmarks that ultimately result in cardiac dysfunction. MicroRNA-25 (miR-25) has been identified as a suppressor of both Sarcoplasmic Reticulum Calcium ATPase 2a (SERCA2a) and Mothers Against Decapentaplegic Homolog-7
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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
Epigenetic processes involving long non-coding RNAs (lncRNAs) regulate endothelial genes expression. However, the underlying regulatory mechanisms causing endothelial dysfunction yet remain to be elucidated. nhancer of este omologue 2 (EZH2) is an important rheostat of histone H3K27 trimethylation (H3K27me3) that represses endothelial targets but EZH2 RNA binding capacity and EZH2:RNA functional interactions
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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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The mRNACalc webserver accounts for the hypochromicity of modified nucleosides and enables the accurate quantification of nucleoside-modified mRNA 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
Nucleoside-modified messenger RNA (mRNA) technologies necessarily incorporate N1-methylpseudouridine into the mRNA molecules to prevent the over-stimulation of cytoplasmic RNA sensors. Despite this modification, mRNA concentrations remain mostly determined through the measurement of UV absorbance at 260 nm wavelength (A). Herein, we report that the N1-methylpseudouridine absorbs approximately 40% less
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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
Duchenne muscular dystrophy (DMD) is the most prevalent herediatry disease in men, characterized by dystrophin deficiency, progressive muscle wasting, cardiac insufficiency, and premature mortality, with no effective therapeutic options. Here, we investigated whether adenine base editing can correct pathological nonsense point mutations leading to premature stop codons in the dystrophin gene. We identified
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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
Transforming growth factor β 1 (TGF-β1), as the most abundant signaling molecule in bone matrix, is essential for bone homeostasis. However, the signaling transduction of TGF-β1 in the bone-forming microenvironment remains unknown. Here, we showed that microRNA-191 (miR-191) was downregulated during osteogenesis and further decreased by osteo-favoring TGF-β1 in bone marrow mesenchymal stem cells (BMSCs)
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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
Anorectal malformations (ARMs) are congenital diseases that lead to postoperative fecal incontinence, constipation, and soiling, despite improvements in surgery; however, their pathological mechanisms remain unclear. Here, we report the role of microRNA-141-3p in maintaining homeostasis between apoptosis and autophagy in the lumbosacral defecation center of fetal rats with ARMs. Elevated microRNA-141-3p
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Trans-amplifying RNA expressing functional miRNA mediates target-specific 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
The co-delivery of microRNAs (miRNAs) and protein-coding RNA presents an opportunity for a combined approach to gene expression and gene regulation for therapeutic applications. Protein delivery is established using long mRNA, self-, and -amplifying RNA (taRNA), whereas miRNA delivery typically uses short synthetic oligonucleotides rather than incorporating it as a precursor into long RNA. Although
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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 M, i, r, o, n, , S, o, p, i, c
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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 C, y, n, t, h, i, a, , D, ., , A, n, d, e, r, s, o, n
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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
(Mtb) infection is among the world’s deadliest infectious diseases. Developing effective treatments and biomarkers for tuberculosis requires a deeper understanding of its pathobiology and host responses. Here, we report a comprehensive characterization of circulating short non-coding RNAs (sncRNAs) in plasma samples from Mtb-infected patients. We achieved this by pre-treating plasma RNAs with T4 polynucleotide
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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
Cisplatin is a highly effective chemotherapeutic agent, but it can cause sensorineural hearing loss (SNHL) in patients. Cisplatin-induced ototoxicity is closely related to the accumulation of reactive oxygen species (ROS) and subsequent death of hair cells (HCs) and spiral ganglion neurons (SGNs). Despite various strategies to combat ototoxicity, only one therapeutic agent has thus far been clinically
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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
New therapies and vaccines based on nucleic acids combined with an efficient nanoparticle delivery vehicle have a broad applicability for different disease indications. An alternative delivery technology for the successfully applied lipid nanoparticles in mRNA SARS-CoV-2 vaccines are nanoparticles composed of biodegradable poly(amido)amine-based polymers with mRNA payload. To show that these polymeric
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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 A, d, a, m, , A, s, t, r, a, d, a
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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
Male infertility has emerged as a global issue, partly attributed to psychological stress. However, the cellular and molecular mechanisms underlying the adverse effects of psychological stress on male reproductive function remain elusive. We created a psychologically stressed model using terrified-sound and profiled the testes from stressed and control rats using single-cell RNA sequencing. Comparative
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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
Endometrial cancer (EC), the second most common malignancy in the female reproductive system, has garnered increasing attention for its genomic heterogeneity, but understanding of its metabolic characteristics is still poor. We explored metabolic dysfunctions in EC through a comprehensive multi-omics analysis (RNA-seq datasets from The Cancer Genome Atlas [TCGA], Cancer Cell Line Encyclopedia [CCLE]
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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
Patients with pre-existing medical conditions are at a heightened risk of contracting severe acute respiratory syndrome (SARS), SARS-CoV-2, and influenza viruses, which can result in more severe disease progression and increased mortality rates. Nevertheless, the molecular mechanism behind this phenomenon remained largely unidentified. Here, we found that microRNA-19a/b (miR-19a/b), which is a constituent
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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
Antisense oligonucleotides (ASOs) were the first modality to pioneer targeted gene knockdown in the treatment of amyotrophic lateral sclerosis (ALS) caused by mutant superoxide dismutase 1 (SOD1). RNA interference (RNAi) is another mechanism of gene silencing in which short interfering RNAs (siRNAs) effectively degrade complementary transcripts. However, delivery to extrahepatic tissues like the CNS
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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, Tassos Georgiadis, James Bainbridge, Michel Michaelides, Almudena Sacristan-Reviriego, Pedro R.L. Perdigão, Amy Leung, Jacqueline van der Spuy
Biallelic variations in the () gene cause Leber congenital amaurosis subtype 4 (LCA4), an autosomal recessive early-onset severe retinal dystrophy that leads to the rapid degeneration of retinal photoreceptors and the severe impairment of sight within the first few years of life. Currently, there is no treatment or cure for -associated LCA4. In this study, we investigated the potential of adeno-associated
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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
Osteogenesis imperfecta (OI) is a rare genetic disease characterized by bone fragility and bone formation. Sclerostin could negatively regulate bone formation by antagonizing the Wnt signal pathway, whereas it imposes severe cardiac ischemic events in clinic. Our team has screened an aptamer that could promote bone anabolic potential without cardiovascular risk. However, the affinity of the aptamer
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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
Endolysins are bacteriophage-encoded hydrolases that show high antibacterial activity and a narrow substrate spectrum. We hypothesize that an mRNA-based approach to endolysin therapy can overcome some challenges of conventional endolysin therapy, namely organ targeting and bioavailability. We show that synthetic mRNA applied to three human cell lines (HEK293T, A549, HepG2 cells) leads to expression
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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
MicroRNA-128-3p (miR-128-3p) is a versatile molecule with multiple functions in the physiopathology of the human central nervous system. Perturbations of miR-128-3p, which is enriched in the brain, contribute to a plethora of neurodegenerative disorders, brain injuries, and malignancies, as this miRNA is a crucial regulator of gene expression in the brain, playing an essential role in the maintenance
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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
MicroRNAs (miRNAs) are involved in post-transcriptional gene expression regulation and in mechanisms of cancer growth and metastases. In this light, miRNAs could be promising therapeutic targets and biomarkers in clinical practice. Therefore, we investigated if specific miRNAs and their target genes contribute to laryngeal squamous cell carcinoma (LSCC) development. We found a significant decrease
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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 M, a, r, c, , G, ü, e, l, l
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
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Immune tolerance promotion by LSEC-specific lentiviral vector-mediated expression of the transgene regulated by the stabilin-2 promoter Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-17 Ester Borroni, Chiara Borsotti, Roberta A. Cirsmaru, Vakhtang Kalandadze, Rosella Famà, Simone Merlin, Brian Brown, Antonia Follenzi
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C-Myc/H19/miR-29b axis downregulates nerve/glial (NG)2 expression in glioblastoma multiforme Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-12 Anne S. Boewe, Selina Wrublewsky, Jessica Hoppstädter, Claudia Götz, Alexandra K. Kiemer, Michael D. Menger, Matthias W. Laschke, Emmanuel Ampofo
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Engineering lentivirus envelope VSV-G for liver targeted delivery of IDOL-shRNA to ameliorate hypercholesterolemia and atherosclerosis Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-11 Wei Wang, Xuemei Chen, Jiali Chen, Menglong Xu, Ying Liu, Shijie Yang, Wenfeng Zhao, Shuhua Tan
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The function of tRNA-derived small RNAs in cardiovascular diseases Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-10 Yan Zhao, Kai Wang, Chun Zhao, Ning Liu, Zhihong Wang, Wenting Yang, Zewei Cheng, Luyu Zhou, Kun Wang
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MicroRNA-centered theranostics for pulmoprotection in critical COVID-19 Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-10 Manel Perez-Pons, Marta Molinero, Iván D. Benítez, María C. García-Hidalgo, Shambhabi Chatterjee, Christian Bär, Jessica González, Antoni Torres, Ferran Barbé, David de Gonzalo-Calvo, CIBERESUCICOVID Project (COV20/00110 ISCIII)
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Multispecies-targeting siRNAs for the modulation of JAK1 in the skin Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-09 Qi Tang, Katherine Y. Gross, Hassan H. Fakih, Samuel O. Jackson, Mohammad Zain U.I. Abideen, Kathryn R. Monopoli, Carine Blanchard, Claire Bouix-Peter, Thibaud Portal, John E. Harris, Anastasia Khvorova, Julia F. Alterman
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DNA electroporation in a vacuum: A “shocking” innovation for vaccines Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-05 Noelia Silva-Pilipich, Aritz Lasarte-Cía, Juan José Lasarte, Cristian Smerdou
Abstract not available
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Targeted nucleic acid delivery for traumatic brain injury: Overcoming blood-brain barrier challenges Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2024-01-02 Jiayu Zhang, Jun Chen
Abstract not available
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miRNAs to the rescue: Reversing heart failure by targeting miR-29 Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2023-12-28 Xinghua Wang, Iqra Anwar, Conrad P. Hodgkinson
Abstract not available
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The regulator of regulators: Understanding the mechanisms behind long noncoding RNA dysregulation Mol. Ther. Nucl. Acids (IF 8.8) Pub Date : 2023-12-28 Z, h, e, n, , Y, a, n, g
Abstract not available