Department of Clinical Neurosciences (DNC), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne University Hospital (CHUV) and University of Lausanne (UNIL), Lausanne Switzerland.
Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne University Hospital (CHUV) and University of Lausanne (UNIL), Lausanne Switzerland.
Hum Gene Ther. 2022 Sep;33(17-18):913-922. doi: 10.1089/hum.2022.177.
The recent success of first central nervous system gene therapies has reinvigorated the growing community of gene therapy researchers and strengthened the field's market position. We are witnessing an increase of clinical trials with long-term efficiency mainly for neurometabolic, neurodegenerative, and neurodevelopmental diseases caused by loss-of-function mutations. The ever-expanding knowledge and accessibility to the most advanced tools allow enrichment of applications to more complex diseases. This gradually contributes toward sealing the gap between top diseases impacting current global health and those toward which gene therapy development is currently aimed. In this study, we highlight innovative therapeutic approaches that have reached the clinics and outline the latest improvements of vector design and targeting. Finally, we address the pressing challenges faced by clinical trials and the direction they are heading.
最近中枢神经系统基因治疗的成功，重新激发了越来越多的基因治疗研究人员的积极性，并巩固了该领域的市场地位。我们正在见证更多临床试验的出现，这些试验主要针对由功能丧失突变引起的神经代谢、神经退行性和神经发育疾病，具有长期疗效。不断扩展的知识和对最先进工具的可及性，使应用范围扩大到更复杂的疾病。这逐渐缩小了当前全球健康所面临的重大疾病与基因治疗发展目标之间的差距。在本研究中，我们重点介绍了已进入临床的创新治疗方法，并概述了载体设计和靶向的最新进展。最后，我们讨论了临床试验所面临的紧迫挑战及其发展方向。
