用于基因治疗的病毒载体鞘内递送

Intrathecal Delivery of Viral Vectors for Gene Therapy.

作者信息

Kagiava Alexia, Kleopa Kleopas A

机构信息

Neuroscience Laboratory, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, Nicosia, Cyprus.

Neurology Clinics, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, Nicosia, Cyprus.

出版信息

Methods Mol Biol. 2018;1791:277-285. doi: 10.1007/978-1-4939-7862-5_22.

DOI:10.1007/978-1-4939-7862-5_22

PMID:30006718

Abstract

Gene delivery to the peripheral nervous system for therapeutic applications remains technically challenging but could eventually have a significant impact on the development of innovative treatments not only for inherited but also for acquired peripheral neuropathies. Here we describe the method for lumbar intrathecal injection of viral vectors in experimental mice. This gene delivery route provides widespread and stable over time Schwann cell-targeted or ubiquitous gene expression in the peripheral nervous system.

摘要

将基因递送至外周神经系统用于治疗应用在技术上仍然具有挑战性，但最终可能不仅对遗传性外周神经病变，而且对获得性外周神经病变的创新治疗发展产生重大影响。在此，我们描述了在实验小鼠中进行腰椎鞘内注射病毒载体的方法。这种基因递送途径可在外周神经系统中随着时间推移提供广泛且稳定的靶向施万细胞或普遍存在的基因表达。