Pharmacodelivery Group, School of Pharmacy, University College Cork, Ireland.
Drug Discov Today. 2011 Mar;16(5-6):203-18. doi: 10.1016/j.drudis.2011.01.003. Epub 2011 Jan 22.
The future of nucleic acid-based therapeutics is dependent on achieving successful delivery. Recently, there has been an increasing interest in delivery via the gastrointestinal tract. Gene therapy via this route has many advantages, including non-invasive access and the versatility to treat local diseases, such as inflammatory bowel disease, as well as systemic diseases, such as haemophilia. However, the intestine presents several distinct barriers and, therefore, the design of robust non-viral delivery systems is key to future success. Several non-viral delivery strategies have provided evidence of activity in vivo. To facilitate the design of more efficient and safe gene medicines, more physiologically relevant models, at both the in vitro and in vivo levels, are essential.
核酸治疗药物的未来取决于能否实现成功递送。最近，人们对通过胃肠道进行递呈的兴趣日益增加。通过该途径进行基因治疗具有许多优势，包括非侵入性进入以及治疗局部疾病（如炎症性肠病）和系统疾病（如血友病）的多功能性。然而，肠道存在几个明显的障碍，因此，设计稳健的非病毒递送系统是未来成功的关键。几种非病毒递送策略已在体内提供了活性证据。为了促进更有效和安全的基因药物的设计，在体外和体内水平上，更需要生理相关的模型。
