体内基因治疗结果转化成像的最新进展。

IF 12.1 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Molecular Therapy Pub Date : 2024-12-30 DOI:10.1016/j.ymthe.2024.12.049
Isabel L Day, Mikayla Tamboline, Gerald S Lipshutz, Shili Xu
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引用次数: 0

摘要

基因疗法通过将包装在运载工具内的遗传物质运送到有缺陷基因的靶细胞中来实现治疗效果。这种方法在治疗各种疾病,包括癌症、代谢紊乱和组织退行性疾病方面显示出了希望。在过去的五年中,分子成像在临床前和临床研究中越来越多地支持基因治疗的发展。来自正电子发射断层扫描(PET)、单光子发射计算机断层扫描(SPECT)、磁共振成像(MRI)和计算机断层扫描(CT)的高质量图像使基因治疗的定量和可靠监测成为可能。大多数已报道的研究已将成像生物标志物应用于非侵入性评估基因治疗的结果。本文旨在为分子影像学和基因治疗的研究人员提供有关这两个学科整合的信息。我们强调了使用成像生物标志物来监测体内基因治疗结果的最新进展,在体内基因治疗中,治疗递送载体是系统管理的。此外,我们讨论了进一步纳入成像生物标志物以支持基因治疗的发展和应用的前景。
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Recent developments in translational imaging of in vivo gene therapy outcomes.

Gene therapy achieves therapeutic benefits by delivering genetic materials, packaged within a delivery vehicle, to target cells with defective genes. This approach has shown promise in treating various conditions, including cancer, metabolic disorders, and tissue-degenerative diseases. Over the past 5 years, molecular imaging has increasingly supported gene therapy development in both preclinical and clinical studies. High-quality images from positron emission tomography (PET), single-photon emission computed tomography (SPECT), magnetic resonance imaging (MRI), and computed tomography (CT) enable quantitative and reliable monitoring of gene therapy. Most reported studies have applied imaging biomarkers to non-invasively evaluate the outcomes of gene therapy. This review aims to inform researchers in molecular imaging and gene therapy about the integration of these two disciplines. We highlight recent developments in using imaging biomarkers to monitor the outcome of in vivo gene therapy, where the therapeutic delivery vehicle is administered systemically. In addition, we discuss prospects for further incorporating imaging biomarkers to support the development and application of gene therapy.

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来源期刊
Molecular Therapy
Molecular Therapy 医学-生物工程与应用微生物
CiteScore
19.20
自引率
3.20%
发文量
357
审稿时长
3 months
期刊介绍: Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.
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