{"title":"Multi-Omics approaches in gene therapy for vascular diseases: bridging genomics, transcriptomics, and epigenetics.","authors":"Jianxin Dong, Ming Sun, Yanmei Li, Zhilei Xie","doi":"10.1080/1061186X.2025.2544786","DOIUrl":null,"url":null,"abstract":"<p><p>Vascular diseases such as atherosclerosis, aneurysms, and peripheral arterial disease remain leading causes of morbidity and mortality, with current treatments primarily managing symptoms rather than addressing underlying molecular drivers. Gene therapy offers a promising avenue for targeted intervention, and recent advances in multi-omics approaches-including genomics, transcriptomics, and epigenetics-are enhancing the precision and efficacy of these therapies. High-throughput sequencing and integrative omics analyses have facilitated the identification of causal genes, non-coding RNAs, and epigenetic regulators involved in vascular pathology. This review examines how multi-omics frameworks inform gene therapy design, from genomic editing of cardiovascular disease loci to transcriptome-guided RNA therapies and epigenetic modulation of disease states. We highlight emerging applications such as CRISPR-based interventions, RNA therapeutics, and individualised precision medicine strategies. Additionally, we address analytical challenges, implementation hurdles, and ethical considerations in translating multi-omics-driven gene therapies into clinical practice. By integrating systems biology and advanced computational methods, the convergence of multi-omics and gene therapy holds transformative potential for vascular medicine, offering new avenues for disease modification and patient-specific therapeutic solutions.</p>","PeriodicalId":15573,"journal":{"name":"Journal of Drug Targeting","volume":" ","pages":"1-16"},"PeriodicalIF":3.9000,"publicationDate":"2025-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Drug Targeting","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/1061186X.2025.2544786","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0
Abstract
Vascular diseases such as atherosclerosis, aneurysms, and peripheral arterial disease remain leading causes of morbidity and mortality, with current treatments primarily managing symptoms rather than addressing underlying molecular drivers. Gene therapy offers a promising avenue for targeted intervention, and recent advances in multi-omics approaches-including genomics, transcriptomics, and epigenetics-are enhancing the precision and efficacy of these therapies. High-throughput sequencing and integrative omics analyses have facilitated the identification of causal genes, non-coding RNAs, and epigenetic regulators involved in vascular pathology. This review examines how multi-omics frameworks inform gene therapy design, from genomic editing of cardiovascular disease loci to transcriptome-guided RNA therapies and epigenetic modulation of disease states. We highlight emerging applications such as CRISPR-based interventions, RNA therapeutics, and individualised precision medicine strategies. Additionally, we address analytical challenges, implementation hurdles, and ethical considerations in translating multi-omics-driven gene therapies into clinical practice. By integrating systems biology and advanced computational methods, the convergence of multi-omics and gene therapy holds transformative potential for vascular medicine, offering new avenues for disease modification and patient-specific therapeutic solutions.
期刊介绍:
Journal of Drug Targeting publishes papers and reviews on all aspects of drug delivery and targeting for molecular and macromolecular drugs including the design and characterization of carrier systems (whether colloidal, protein or polymeric) for both vitro and/or in vivo applications of these drugs.
Papers are not restricted to drugs delivered by way of a carrier, but also include studies on molecular and macromolecular drugs that are designed to target specific cellular or extra-cellular molecules. As such the journal publishes results on the activity, delivery and targeting of therapeutic peptides/proteins and nucleic acids including genes/plasmid DNA, gene silencing nucleic acids (e.g. small interfering (si)RNA, antisense oligonucleotides, ribozymes, DNAzymes), as well as aptamers, mononucleotides and monoclonal antibodies and their conjugates. The diagnostic application of targeting technologies as well as targeted delivery of diagnostic and imaging agents also fall within the scope of the journal. In addition, papers are sought on self-regulating systems, systems responsive to their environment and to external stimuli and those that can produce programmed, pulsed and otherwise complex delivery patterns.