Batty Paul, Lillicrap David
Department of Pathology & Molecular Medicine, Richardson Laboratory, Queen's University, Kingston, Ontario, Canada.
Hemasphere. 2021 Feb 10;5(3):e540. doi: 10.1097/HS9.0000000000000540. eCollection 2021 Mar.
The clinical potential of hemophilia gene therapy has now been pursued for the past 30 years, and there is a realistic expectation that this goal will be achieved within the next couple of years with the licensing of a gene therapy product. While recent late phase clinical trials of hemophilia gene therapy have shown promising results, there remain a number of issues that require further attention with regard to both efficacy and safety of this therapeutic approach. In this review, we present information relating to the current status of the field and focus attention on the unanswered questions for hemophilia gene therapy and the future challenges that need to be overcome to enable the widespread application of this treatment paradigm.
在过去的30年里，血友病基因治疗的临床潜力一直在被探索，人们切实期望在未来几年内随着一种基因治疗产品获得许可，这一目标将得以实现。虽然最近血友病基因治疗的晚期临床试验已显示出令人鼓舞的结果，但在这种治疗方法的有效性和安全性方面，仍有一些问题需要进一步关注。在本综述中，我们介绍了该领域的现状，并重点关注血友病基因治疗中尚未解决的问题以及为使这种治疗模式得以广泛应用而需要克服的未来挑战。
