Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain.
Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain.
Hum Gene Ther. 2023 Sep;34(17-18):763-775. doi: 10.1089/hum.2023.152.
The development of viral vectors and recombinant DNA technology since the 1960s has enabled gene therapy to become a real therapeutic option for several inherited and acquired diseases. After several ups and downs in the gene therapy field, we are currently living a new era in the history of medicine in which several and gene therapies have reached maturity. This is testified by the recent marketing authorization of several gene therapy medicinal products. In addition, many others are currently under evaluation after exhaustive investigation in human clinical trials. In this review, we summarize some of the most significant milestones in the development of gene therapy medicinal products that have already facilitated the treatment of a significant number of rare diseases. Despite progresses in the gene therapy field, the transfer of these innovative therapies to clinical practice is also finding important restrictions. Advances and also challenges in the progress of gene therapy for rare diseases are discussed in this opening review of a issue dedicated to the 30th annual Congress of the European Society for Gene and Cell Therapy.
自 20 世纪 60 年代以来，病毒载体和重组 DNA 技术的发展使基因治疗成为几种遗传性和获得性疾病的真正治疗选择。在基因治疗领域经历了几次起伏之后，我们目前正处于医学史上的一个新时代，几种基因和细胞疗法已经成熟。这一点可以通过最近几种基因治疗药物的营销授权得到证明。此外，在经过人类临床试验的详尽研究后，还有许多其他基因治疗药物正在评估中。在这篇综述中，我们总结了已经促成了大量罕见疾病治疗的一些最重要的基因治疗药物开发里程碑。尽管基因治疗领域取得了进展，但这些创新疗法在向临床实践的转移中也发现了重要的限制。在这篇对基因和细胞治疗学会第 30 届年会的一个专刊的开篇综述中，讨论了罕见病基因治疗的进展和挑战。
