神经成像在中枢神经系统疾病基因治疗的传递和监测中的应用。

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Human gene therapy Pub Date : 2024-11-01 Epub Date: 2024-11-08 DOI:10.1089/hum.2024.057
Rrita Daci, Heather Gray-Edwards, Mohammed Salman Shazeeb, Zeynep Vardar, Behroze Vachha, Oguz I Cataltepe, Terence R Flotte
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引用次数: 0

摘要

单基因缺陷导致的神经系统疾病是腺相关病毒(AAV)介导的基因疗法的一个靶点。由于存在血脑屏障,将 AAV 介导的基因疗法输送到大脑具有挑战性。基因转移技术,如对流增强的实质内转移和图像引导的大脑脑脊液(CSF)空间转移,已将该领域引向高精度的转移技术,从而纠正特定脑区或更广泛范围内的基因缺陷。这些技术通常使用磁共振成像(MRI)、计算机断层扫描(CT)和透视引导。此外,核磁共振成像(MRI)、核磁共振波谱成像(MRS)、弥散张量成像(DTI)和功能核磁共振成像(fMRI)所评估的神经影像变化可作为治疗效果和整体疾病进展的重要生物标志物。在此,我们将讨论神经成像在递送 AAV 向量和监测基因治疗效果中的作用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases.

Neurological disease due to single-gene defects represents a targetable entity for adeno-associated virus (AAV)-mediated gene therapy. The delivery of AAV-mediated gene therapy to the brain is challenging, owing to the presence of the blood-brain barrier. Techniques in gene transfer, such as convection-enhanced intraparenchymal delivery and image-guided delivery to the cerebrospinal fluid spaces of the brain, have led the field into highly accurate delivery techniques, which provide correction of genetic defects in specific brain regions or more broadly. These techniques commonly use magnetic resonance imaging (MRI), computed tomography, and fluoroscopic guidance. Even more, the neuroimaging changes evaluated by MRI, MR spectroscopy, diffusion tensor imaging, and functional MRI can serve as important biomarkers of therapy effect and overall disease progression. Here, we discuss the role of neuroimaging in delivering AAV vectors and monitoring the effect of gene therapy.

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来源期刊
Human gene therapy
Human gene therapy 医学-生物工程与应用微生物
CiteScore
6.50
自引率
4.80%
发文量
131
审稿时长
4-8 weeks
期刊介绍: Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
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