Schimmer Joshua, Breazzano Steven
Piper Jaffray & Co. , New York, New York.
Hum Gene Ther Clin Dev. 2016 Sep;27(3):87-92. doi: 10.1089/humc.2016.29017.ind.
The gene therapy field continues to pick up steam with recent successes in a number of different therapeutic indications that highlight the potential for the platform. As the field continues to make progress, a growing data set of long-term safety and efficacy data will continue to define gene therapy's role, determining ultimately how widely it may be used beyond rare, serious diseases with high unmet needs. New technologies often take unanticipated twists and turns as patient exposure accumulates, and gene therapy may be no exception. That said, with many diseases that have no other treatment options beyond gene therapy and that present considerable morbidity and mortality, the field appears poised to withstand some minor and even major bumps in the road should they emerge.
随着基因疗法在许多不同治疗适应症方面最近取得成功，该领域的发展势头持续增强，凸显了该平台的潜力。随着该领域不断取得进展，越来越多的长期安全性和有效性数据将继续界定基因疗法的作用，最终决定它在罕见、严重且需求未得到充分满足的疾病之外能够在多大范围内得到广泛应用。随着患者接触量的积累，新技术往往会出现意想不到的波折，基因疗法可能也不例外。话虽如此，对于许多除了基因疗法之外别无其他治疗选择且具有相当高发病率和死亡率的疾病而言，即便该领域在发展过程中出现一些小的甚至大的波折，它似乎也有能力承受。
