Christ George J
Wake Forest Institute for Regenerative Medicine, Wake Forest University School of Medicine Baptist Medical Center, Winston-Salem, NC, USA.
Handb Exp Pharmacol. 2011(202):255-65. doi: 10.1007/978-3-642-16499-6_12.
Identification of molecular targets for novel therapeutics is a natural consequence of the age of molecular and personalized medicine. How this information is leveraged and applied to the treatment of functional diseases/disorders of the lower urinary tract will determine if this field of medicine can keep pace with technological developments and patient expectations for improved therapies. In this regard, therapeutic improvements for the treatment of lower urinary tract diseases and disorders have been largely incremental over the past 30 years. The goal of this report is to review the evidence pointing toward the enormous potential of gene therapy/transfer to provide a paradigm shift from palliative to curative therapeutic solutions for lower urinary tract diseases/disorders. In fact, it seems clear that gene therapy represents a biotechnology approach particularly suitable to applications in the lower urinary tract. Although much more research is required, ample preclinical evidence already indicates that, for example, gene therapy can favorably impact/alter virtually every aspect of bladder physiology/function. In short, further investigations and continued applications of gene therapy to the treatment of lower urinary tract diseases/disorders seems a prudent step toward potentially marked and more durable therapeutic improvements.
识别新型治疗方法的分子靶点是分子医学和个性化医学时代的自然产物。如何利用这些信息并将其应用于下尿路功能性疾病的治疗，将决定该医学领域能否跟上技术发展以及患者对改进疗法的期望。在这方面，过去30年来，下尿路疾病治疗方法的改进在很大程度上是渐进式的。本报告的目的是回顾相关证据，这些证据表明基因治疗/基因转移具有巨大潜力，可为下尿路疾病提供从姑息治疗到治愈性治疗方案的范式转变。事实上，基因治疗似乎显然是一种特别适用于下尿路应用的生物技术方法。尽管还需要更多研究，但大量临床前证据已经表明，例如，基因治疗几乎可以对膀胱生理/功能的各个方面产生有利影响/改变。简而言之，进一步研究并持续将基因治疗应用于下尿路疾病的治疗，似乎是朝着实现潜在的显著且更持久的治疗改进迈出的谨慎一步。
