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Molecular Therapy. Nucleic Acids最新文献

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Retraction Notice to: circFLT1 and lncCCPG1 Sponges miR-93 to Regulate the Proliferation and Differentiation of Adipocytes by Promoting lncSLC30A9 Expression. 关于circFLT1和lncCCPG1海绵miR-93通过促进lnccslc30a9的表达来调节脂肪细胞的增殖和分化的撤回通知

IF 6.1 2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-03-04 eCollection Date: 2026-03-12 DOI: 10.1016/j.omtn.2026.102877

Zihong Kang, Sihuang Zhang, Enhui Jiang, Xinyu Wang, Zhen Wang, Hong Chen, Xianyong Lan

引用次数: 0

Retraction Notice to: circFGFR4 Promotes Differentiation of Myoblasts via Binding miR-107 to Relieve Its Inhibition of Wnt3a. 撤回通知：circFGFR4通过结合miR-107促进成肌细胞分化，减轻其对Wnt3a的抑制。

IF 6.1 2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-03-04 eCollection Date: 2026-03-12 DOI: 10.1016/j.omtn.2026.102876

Hui Li, Xuefeng Wei, Jiameng Yang, Dong Dong, Dan Hao, Yongzhen Huang, Xianyong Lan, Martin Plath, Chuzhao Lei, Yun Ma, Fengpeng Lin, Yueyu Bai, Hong Chen

引用次数: 0

Enhanced liver-targeted delivery with ribofuranose-based GalNAc conjugates. 基于核糖呋喃糖的GalNAc缀合物增强肝脏靶向递送。

IF 6.1 2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-03-04 eCollection Date: 2026-03-12 DOI: 10.1016/j.omtn.2026.102871

Sai Pallavi Pradeep, Ruchi Ruchi, Raman Bahal

{"title":"Enhanced liver-targeted delivery with ribofuranose-based GalNAc conjugates.","authors":"Sai Pallavi Pradeep, Ruchi Ruchi, Raman Bahal","doi":"10.1016/j.omtn.2026.102871","DOIUrl":"https://doi.org/10.1016/j.omtn.2026.102871","url":null,"abstract":"<p><p>This study highlights that small geometric constraints with the GalNAc moiety can tune receptor engagement and serum stability. The ribofuranose ring is a 2'-O-methyl modification, used in the siRNA backbone to improve metabolic stability and reduce nuclease susceptibility.<sup>8</sup> The constrained ribofuranose conformation introduced into GalNAc increased serum stability and hepatic parenchymal clearance, thereby increasing systemic exposure. They also demonstrate the kilogram-scale synthesis of the G5 GalNAc-CPG support, making it feasible for manufacturing. PCSK9 is an ideal benchmark target as it is clinically validated, and the observed performance can be applied to other hepatocyte-expressed genes. The inc-G5 GalNAc has progressed to phase 1 trials in China for PCSK9- and AGT-targeting siRNA. In a non-human primate study, inc-G5 GalNAc-siRNA conjugates showed a safety profile consistent with established GalNAc-siRNA (inc-L96) conjugates. A comparison of toxicology in AGT-targeting siRNA versus zilebesiran showed similar clinical chemistry and histopathology, supporting inc-G5 GalNAc as a potent, clinically translatable GalNAc delivery scaffold.</p>","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"37 1","pages":"102871"},"PeriodicalIF":6.1,"publicationDate":"2026-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12969788/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147434271","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Retraction Notice to: Circular RNA circMYBPC1 promotes skeletal muscle differentiation by targeting MyHC. 环状RNA circMYBPC1通过靶向MyHC促进骨骼肌分化。

IF 6.1 2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-03-03 eCollection Date: 2026-03-12 DOI: 10.1016/j.omtn.2026.102875

Mengjie Chen, Xuefeng Wei, Mingming Song, Rui Jiang, Kongwei Huang, Yanfei Deng, Qingyou Liu, Deshun Shi, Hui Li

引用次数: 0

Advancing the science of nucleic acids together. 共同推进核酸科学。

IF 6.1 2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-03-02 eCollection Date: 2026-03-12 DOI: 10.1016/j.omtn.2026.102861

Betty Schiefelbein, Paloma H Giangrande, Zhijian Chen

引用次数: 0

TIPE2 Gene Knockdown in Mice Attenuates Experimental Colitis by Diminishing Inflammatory Cell Infiltration TIPE2基因敲低可通过减少炎性细胞浸润减轻实验性结肠炎

2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-03-01 DOI: 10.1016/j.omtn.2026.102887

Yexiao Tang, Ping Li, Yajie Lu, Pengchao Zhang, Zhen Lu, Hang Qiao, Jing Wei, Zhiming Xu, X. Steven Wan, Shu Xu, Youhai H. Chen, Guizhong Zhang

引用次数: 0

An inflammation-defying adjuvant "micro"manages age-diminished vaccine efficacy. 抗炎佐剂“微”管理年龄降低的疫苗效力。

IF 6.1 2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-02-23 eCollection Date: 2026-03-12 DOI: 10.1016/j.omtn.2026.102864

GuanQun Liu

引用次数: 0

miRNA675-5p inhibitor's dual role as novel therapeutic alternative or sensitizing treatment in resistant glioma models. miRNA675-5p抑制剂在耐药胶质瘤模型中作为新的治疗选择或增敏治疗的双重作用。

IF 6.1 2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-02-18 eCollection Date: 2026-03-12 DOI: 10.1016/j.omtn.2025.102647

Cristina Martelli, Marcella Bonanomi, Chiara Pellizzer, Alessandro Giammona, Sofia Remedia, Martina Nespoli, Daniela Gaglio, Daniele Capitanio, Danilo Porro, Luisa Ottobrini, Alessia Lo Dico

{"title":"miRNA675-5p inhibitor's dual role as novel therapeutic alternative or sensitizing treatment in resistant glioma models.","authors":"Cristina Martelli, Marcella Bonanomi, Chiara Pellizzer, Alessandro Giammona, Sofia Remedia, Martina Nespoli, Daniela Gaglio, Daniele Capitanio, Danilo Porro, Luisa Ottobrini, Alessia Lo Dico","doi":"10.1016/j.omtn.2025.102647","DOIUrl":"10.1016/j.omtn.2025.102647","url":null,"abstract":"<p><p>Glioma is currently the most aggressive CNS tumor. MicroRNA (miRNA)675-5p is a hypoxic miRNA involved in promoting and maintaining the hypoxia inducible factor (HIF)-1α pathway, the driving force for glioma proliferation, migration into surrounding tissue, and resistance. Its inhibition is effective in reducing HIF-1α and all pathways related to it. However, the molecular mechanism through which miRNA inhibition is effective has not yet been fully elucidated. The therapeutic efficacy of the miRNA675-5p inhibitor was tested in a panel of resistant glioma lines evaluating the cellular, molecular, and biochemical rearrangement of the cells after treatment, with particular attention to the oxidative stress imbalance. miRNA675-5p inhibitor has a therapeutic efficacy on its own in resistant cell lines, reducing HIF-1α and its related pathways. The mechanism through which this occurs is the induction of oxidative stress. Its impairment, in fact, reverses the cytotoxic effect. Inhibitor-treated cells acquire metabolic characteristics clearly distinct from untreated cells, triggering compensatory mechanisms that must be considered for the secondary treatment of glioma with temozolomide. The induction of oxidative stress and metabolic rearrangement play key roles in the cytotoxic effect of the miRNA675-5p inhibitor, which could be proposed as a new therapeutic approach in glioma.</p>","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"37 1","pages":"102647"},"PeriodicalIF":6.1,"publicationDate":"2026-02-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12984634/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147463701","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Generation of aptamers for the selective detection and neutralization of soluble lymphotoxin alpha. 选择性检测和中和可溶性淋巴蛋白α的适体的产生。

IF 6.1 2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-02-17 eCollection Date: 2026-03-12 DOI: 10.1016/j.omtn.2026.102867

Matthew Stephens, Eman Nassef, Pierre-Yves von der Weid

{"title":"Generation of aptamers for the selective detection and neutralization of soluble lymphotoxin alpha.","authors":"Matthew Stephens, Eman Nassef, Pierre-Yves von der Weid","doi":"10.1016/j.omtn.2026.102867","DOIUrl":"https://doi.org/10.1016/j.omtn.2026.102867","url":null,"abstract":"<p><p>Lymphotoxin alpha (LTα) is a potent inflammatory cytokine implicated in the pathophysiology of numerous human autoimmune and inflammatory diseases. Existing as a soluble homotrimer (LTα<sub>3</sub>) or membrane-bound heterotrimer (LTα<sub>1</sub>β<sub>2</sub>), the differential and distinct functions of lymphotoxin signaling have meant that selective targeting of the cytokine with traditional pharmacological agents has proven difficult. While monoclonal antibodies that can neutralize human LTα<sub>3</sub> <i>in vivo</i> do exist (e.g., pateclizumab), their efficacy and subsequent use within the clinic have been limited. This may be in part perhaps due to cross-reactivity between the homotrimeric and heterotrimeric forms, leading to LTα<sub>3</sub>-independent effects. Herein, we implement a counter-Systematic Evolution of Ligands by Exponential Enrichment (SELEX) protocol to enrich aptamers targeting LTα<sub>3</sub> but not LTα<sub>1</sub>β<sub>2.</sub> Through a combination of <i>in silico</i> and <i>in vitro</i> tests, we also refine the aptamer sequences and test their ability to limit LT⍺<sub>3</sub>-TNFR1 engagement and subsequent cellular cytotoxicity in L929 fibroblasts. We highlight the generation of 4 aptamer candidates that can selectively detect LTα<sub>3</sub> but not LTα<sub>1</sub>β<sub>2</sub>. Using rational design, we optimize the sequences and show that LTa1 and LTa5 can significantly reduce LTa<sub>3</sub>-TNFR1 engagement-associated cytotoxicity <i>in vitro</i>, highlighting their future therapeutic potential. These data highlight aptamers for the future investigation of lymphotoxin signaling, limiting off-target impacts on other LT⍺<sub>3</sub>-dependent mechanisms.</p>","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"37 1","pages":"102867"},"PeriodicalIF":6.1,"publicationDate":"2026-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12969614/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147434260","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Optimizing C14120-based LNPs for in vitro and in vivo mRNA delivery. 优化基于c14120的LNPs体外和体内mRNA传递。

IF 6.1 2区医学

Molecular Therapy. Nucleic Acids Pub Date : 2026-02-13 eCollection Date: 2026-03-12 DOI: 10.1016/j.omtn.2026.102866

Ping Song, Junyi Su, Camilla Lilly Kristine Vraa, Maria Gockert, Søren Fjelstrup, Henrik Hager, Jørgen Kjems

{"title":"Optimizing C14120-based LNPs for <i>in vitro</i> and <i>in vivo</i> mRNA delivery.","authors":"Ping Song, Junyi Su, Camilla Lilly Kristine Vraa, Maria Gockert, Søren Fjelstrup, Henrik Hager, Jørgen Kjems","doi":"10.1016/j.omtn.2026.102866","DOIUrl":"https://doi.org/10.1016/j.omtn.2026.102866","url":null,"abstract":"<p><p>Lipid nanoparticles (LNPs) have proven to be an effective delivery system for RNA therapeutics. The chemical composition of LNPs determines their functional delivery efficiency and targeting properties, which vary between <i>in vitro</i> and <i>in vivo</i> contexts. Here, we have systematically characterized and compared 25 novel C14120-based LNP formulations for mRNA delivery <i>in vitro</i> and assessed <i>in vivo</i> mRNA expression and biodistribution using deep sequencing of DNA barcodes in a pooled LNP-mRNA library. <i>In vitro</i> experiments showed correlations of lipid composition with particle size and mRNA transfection efficiency in 4 different cell lines of distinct tissue and species origin. <i>In vivo</i> experiments employed a pooled LNP delivery of luciferase mRNA in combination with a multiplexed barcode system and identified LNP compositions with organ-specific targeting properties. Individual validation of three selected LNP candidates based on mRNA expression analysis confirmed high specificity for the lung-targeting candidate, lower specificity for the liver-targeting candidate, and inconclusive results for the spleen-targeting candidate. These findings identify LNP formulations with promising potential for <i>in vitro</i> and <i>in vivo</i> organ-targeted delivery.</p>","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"37 1","pages":"102866"},"PeriodicalIF":6.1,"publicationDate":"2026-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12972518/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147434330","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

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