Haggerty David L, Grecco Gregory G, Reeves Kaitlin C, Atwood Brady
Department of Pharmacology & Toxicology, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Medical Scientist Training Program, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Mol Ther Methods Clin Dev. 2019 Nov 26;17:69-82. doi: 10.1016/j.omtm.2019.11.012. eCollection 2020 Jun 12.
Adeno-associated viral vectors (AAVs) are increasingly useful preclinical tools in neuroscience research studies for interrogating cellular and neurocircuit functions and mapping brain connectivity. Clinically, AAVs are showing increasing promise as viable candidates for treating multiple neurological diseases. Here, we briefly review the utility of AAVs in mapping neurocircuits, manipulating neuronal function and gene expression, and activity labeling in preclinical research studies as well as AAV-based gene therapies for diseases of the nervous system. This review highlights the vast potential that AAVs have for transformative research and therapeutics in the neurosciences.
腺相关病毒载体（AAVs）在神经科学研究中作为用于探究细胞和神经回路功能以及绘制脑连接图谱的临床前工具越来越有用。在临床上，AAVs作为治疗多种神经系统疾病的可行候选物正显示出越来越大的前景。在这里，我们简要回顾AAVs在临床前研究中绘制神经回路、操纵神经元功能和基因表达以及活性标记方面的效用，以及基于AAV的神经系统疾病基因疗法。这篇综述强调了AAVs在神经科学转化研究和治疗方面的巨大潜力。
