Molecular Therapy. Nucleic Acids最新文献

{"title":"The circITSN1/eIF4A3/Itsn1 axis, a potential new molecular entry point for managing postoperative cognitive dysfunction.","authors":"Sedef Ersoy, Christian Bär","doi":"10.1016/j.omtn.2025.102674","DOIUrl":"10.1016/j.omtn.2025.102674","url":null,"abstract":"","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102674"},"PeriodicalIF":6.1,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12447564/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145113795","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}

{"title":"RNA-based therapeutic interventions for the management of Anderson-Fabry disease.","authors":"Stefano Cagnin","doi":"10.1016/j.omtn.2025.102679","DOIUrl":"10.1016/j.omtn.2025.102679","url":null,"abstract":"","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102679"},"PeriodicalIF":6.1,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12408215/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145015885","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}

{"title":"The potential role of microRNA-mediated motor neuron-muscle pathologic interactions in amyotrophic lateral sclerosis.","authors":"Yuji Ueno, Yuki Nakamura, Takanori Hata","doi":"10.1016/j.omtn.2025.102675","DOIUrl":"10.1016/j.omtn.2025.102675","url":null,"abstract":"","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102675"},"PeriodicalIF":6.1,"publicationDate":"2025-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12397938/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144961751","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}

{"title":"Dual inhibition of ACE-2 and TMPRSS2: A promising strategy to mitigate SARS-CoV-2 severity in smokers.","authors":"Kingshuk Panda, Srinivasan Chinnapaiyan, Hoshang J Unwalla","doi":"10.1016/j.omtn.2025.102662","DOIUrl":"10.1016/j.omtn.2025.102662","url":null,"abstract":"","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102662"},"PeriodicalIF":6.1,"publicationDate":"2025-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12397935/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144961867","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}

{"title":"Erratum: RNA activation of <i>CEBPA</i> improves leukemia treatment.","authors":"Olivia Kovecses, Bahram Sharif-Askari, Cristobal Gonzalez-Losada, Vikash Reebye, Bríd M Ryan, Albert Kwok, Nagy A Habib, Nathan W Luedtke, François E Mercier, Maureen McKeague","doi":"10.1016/j.omtn.2025.102643","DOIUrl":"https://doi.org/10.1016/j.omtn.2025.102643","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1016/j.omtn.2025.102611.].</p>","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102643"},"PeriodicalIF":6.1,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12447566/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145113650","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}

{"title":"Targeting long non-coding RNA <i>MALAT1</i> preserves endothelial cell integrity and protects against kidney fibrosis.","authors":"Qiao Zhao, Loïs A K van der Pluijm, Morgane Gourvest, Atefeh Lafzi, Daniel Peled, Whitney G Rubin, Juliette A de Klerk, Roderick C Slieker, Leen M 't Hart, Wendy Stam, Annemarie M van Oeveren-Rietdijk, Jacques M G J Duijs, Angela Koudijs, Joris I Rotmans, Hilal Kazan, Anton Jan van Zonneveld, Coen van Solingen, Roel Bijkerk","doi":"10.1016/j.omtn.2025.102689","DOIUrl":"10.1016/j.omtn.2025.102689","url":null,"abstract":"<p><p>Loss of integrity of the capillary network is directly associated with the development of kidney fibrosis resulting in chronic kidney disease. Here, we characterized long non-coding RNAs (lncRNAs) in endothelial cells (ECs) during the development of kidney fibrosis. Using a murine EC lineage-tracing model, we observed expression of the conserved lncRNA metastasis-associated lung adenocarcinoma transcript 1 (<i>Malat1)</i> to be elevated in ECs upon kidney injury; either by ischemia-reperfusion injury or by unilateral ureteral obstruction (UUO). In addition, we found elevated <i>MALAT1</i> expression in the kidney and circulation of patients with fibrotic kidney diseases. Pharmacological intervention of <i>Malat1</i> initiated protection against fibrosis in the UUO model, illustrated by a marked decline in collagen deposition and a concomitant decrease in interstitial alpha-smooth muscle actin (α-SMA)-positive cells in the kidney. This protective effect was further highlighted by an increase in capillary density and reduced endothelial-to-mesenchymal transition. Mechanistically, transcriptomic analyses of kidney ECs upon <i>Malat1</i> knockdown demonstrated increased EC-matrix-receptor interaction. Furthermore, we show that silencing of <i>MALAT1</i> results in increased barrier function and angiogenic response, less vascular leakage, and decreased focal adhesions. Finally, integration with <i>in silico</i> analyses and RNA immunoprecipitation confirmed binding of <i>MALAT1</i> to SUZ12, a member of the PRC2 complex, suggesting a transcriptional regulatory role for <i>MALAT1</i>. Collectively, our findings classify the lncRNA <i>MALAT1</i> as an important regulator of EC function and kidney health. As such, targeting <i>MALAT1</i> may provide novel strategies to reduce kidney fibrosis.</p>","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102689"},"PeriodicalIF":6.1,"publicationDate":"2025-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12445227/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145113817","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}

{"title":"Minimalist mRNAs: A fast track to cancer vaccines.","authors":"Laura Salaberry, Ángela Covo-Vergara, Uxue Beloki, Noelia Silva-Pilipich, Cristian Smerdou","doi":"10.1016/j.omtn.2025.102669","DOIUrl":"10.1016/j.omtn.2025.102669","url":null,"abstract":"","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102669"},"PeriodicalIF":6.1,"publicationDate":"2025-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12395492/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144961660","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}

{"title":"FOXO4-AR crosstalk in PCOS: A pivotal mechanism of anovulation.","authors":"Shuangning Qian, Guojun Pan, Mingjie Fan","doi":"10.1016/j.omtn.2025.102663","DOIUrl":"10.1016/j.omtn.2025.102663","url":null,"abstract":"","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102663"},"PeriodicalIF":6.1,"publicationDate":"2025-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12391437/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144961877","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}

{"title":"PAI-1 and CHRNA1-A new molecular axis and therapeutic target in primary focal hyperhidrosis.","authors":"Marie Hoareau, Rong-Mo Zhang","doi":"10.1016/j.omtn.2025.102664","DOIUrl":"10.1016/j.omtn.2025.102664","url":null,"abstract":"","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102664"},"PeriodicalIF":6.1,"publicationDate":"2025-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12391439/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144961745","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}

{"title":"Molecular treatment options for patients carrying <i>KIAA0586/TALPID3</i> variants.","authors":"Jacqueline E Taudien, Sebastian Swirski, Maike Möller, Christoph Jüschke, Marta Owczarek-Lipska, G Christoph Korenke, John Neidhardt","doi":"10.1016/j.omtn.2025.102688","DOIUrl":"10.1016/j.omtn.2025.102688","url":null,"abstract":"<p><p>Variants in <i>KIAA0586/TALPID3</i> are associated with the ciliopathy Joubert syndrome (JS), which is a genetically heterogeneous disorder. Mutations in more than 40 different genes were associated with JS, genes that are relevant to ciliary assembly, maintenance, or cellular signaling pathways during the development. Genetic variability suggests that gene- and/or mutation-independent treatment strategies could be beneficial to patients. Currently, targeted therapeutic options are not available. In this study, we present molecular treatment options for pathogenic <i>KIAA0586/TALPID3</i> sequence alterations. We compared therapeutic efficacy and side effects using patient-derived fibroblasts from two siblings affected by JS. The patients harbored two compound heterozygous sequence alterations, a non-sense mutation (<i>KIAA0586/TALPID3</i>: c.2353C>T) in exon 18 and a deep-intronic mutation in intron 28 (<i>KIAA0586/TALPID3</i>: c.3990 + 3186G>A). The deep-intronic sequence alteration activates a cryptic exon that causes a frameshift and splicing defect. Both variants are predicted to potentially result in the premature termination of translation. The patient-derived fibroblasts exhibited reduced primary cilia length and altered distribution of PCM1. These cellular defects were responsive to treatments with RNA-based therapeutics and/or readthrough agents (RTAs). Our results highlight the potential of addressing mutations and molecular defects associated with <i>KIAA0586/TALPID3</i> sequence alterations as future perspectives toward treatments of patients.</p>","PeriodicalId":18821,"journal":{"name":"Molecular Therapy. Nucleic Acids","volume":"36 3","pages":"102688"},"PeriodicalIF":6.1,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12423346/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145065303","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}