Cantore Alessio, Fraldi Alessandro, Meneghini Vasco, Gritti Angela
San Raffaele Telethon Institute for Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy.
School of Medicine, Vita-Salute San Raffaele University, Milan, Italy.
Front Med (Lausanne). 2022 Jan 18;8:774618. doi: 10.3389/fmed.2021.774618. eCollection 2021.
genetic engineering has recently shown remarkable potential as a novel effective treatment for an ever-growing number of diseases, as also witnessed by the recent marketing authorization of several gene therapy products. genetic engineering comprises both viral vector-mediated gene transfer and the more recently developed genome/epigenome editing strategies, as long as they are directly administered to patients. Here we first review the most advanced gene therapies that are commercially available or in clinical development. We then highlight the major challenges to be overcome to fully and broadly exploit gene therapies as novel medicines, discussing some of the approaches that are being taken to address them, with a focus on the nervous system and liver taken as paradigmatic examples.
基因工程最近已显示出巨大潜力，可作为一种新型有效疗法用于治疗越来越多的疾病，这也从最近几种基因治疗产品获得上市许可得到印证。基因工程既包括病毒载体介导的基因转移，也包括最近开发的基因组/表观基因组编辑策略，只要它们是直接施用于患者的。在此，我们首先回顾一下目前已上市或正在临床开发中的最先进的基因疗法。然后，我们将重点强调要全面广泛地将基因疗法作为新型药物加以利用所面临的主要挑战，并讨论为应对这些挑战所采取的一些方法，重点以神经系统和肝脏作为典型例子进行探讨。
