Day Isabel L, Tamboline Mikayla, Lipshutz Gerald S, Xu Shili
Department of Molecular and Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA; Crump Institute for Molecular Imaging, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Department of Molecular and Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Psychiatry and Biobehavioral Sciences, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Surgery, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA; Intellectual and Developmental Disabilities Research Center, University of California, Los Angeles, Los Angeles, CA 90095, USA; Semel Institute for Neuroscience, University of California, Los Angeles, Los Angeles, CA 90095, USA; Molecular Biology Institute, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA; Jonsson Comprehensive Cancer Center, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Mol Ther. 2025 Jun 4;33(6):2548-2564. doi: 10.1016/j.ymthe.2024.12.049. Epub 2024 Dec 30.
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.
基因治疗通过将包装在递送载体中的遗传物质递送至具有缺陷基因的靶细胞来实现治疗益处。这种方法在治疗各种病症方面已显示出前景，包括癌症、代谢紊乱和组织退行性疾病。在过去5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）的高质量图像能够对基因治疗进行定量且可靠的监测。大多数已报道的研究都应用成像生物标志物来非侵入性地评估基因治疗的结果。本综述旨在让分子成像和基因治疗领域的研究人员了解这两个学科的整合情况。我们重点介绍了使用成像生物标志物监测体内基因治疗结果的最新进展，其中治疗性递送载体是全身给药的。此外，我们还讨论了进一步纳入成像生物标志物以支持基因治疗的开发和应用的前景。
