血管疾病基因治疗中的多组学方法:连接基因组学、转录组学和表观遗传学。

IF 3.9 4区 医学 Q1 PHARMACOLOGY & PHARMACY
Jianxin Dong, Ming Sun, Yanmei Li, Zhilei Xie
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引用次数: 0

摘要

动脉粥样硬化、动脉瘤和外周动脉疾病等血管疾病仍然是发病率和死亡率的主要原因,目前的治疗主要是控制症状,而不是解决潜在的分子驱动因素。基因治疗为靶向干预提供了一条很有前途的途径,而多组学方法的最新进展——包括基因组学、转录组学和表观遗传学——正在提高这些治疗的准确性和有效性。高通量测序和整合组学分析有助于确定血管病理相关的致病基因、非编码rna和表观遗传调控因子。这篇综述探讨了多组学框架如何为基因治疗设计提供信息,从心血管疾病基因座的基因组编辑到转录组引导的RNA治疗和疾病状态的表观遗传调节。我们强调了新兴的应用,如基于crispr的干预,RNA治疗和个性化的精准医疗策略。此外,我们解决了在将多组学驱动的基因疗法转化为临床实践中的分析挑战、实施障碍和伦理考虑。通过整合系统生物学和先进的计算方法,多组学和基因治疗的融合为血管医学提供了变革的潜力,为疾病改造和患者特异性治疗解决方案提供了新的途径。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Multi-Omics approaches in gene therapy for vascular diseases: bridging genomics, transcriptomics, and epigenetics.

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.

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来源期刊
CiteScore
9.10
自引率
0.00%
发文量
165
审稿时长
2 months
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.
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