Gene Therapy Center, University of North Carolina at Chapel Hill, 7109 Thurston Bowles, 104 Manning Drive, Chapel Hill, NC 27599-7352, USA.
Neuropharmacology. 2013 May;68:136-42. doi: 10.1016/j.neuropharm.2012.06.024. Epub 2012 Jun 27.
With a number of recent clinical successes, gene therapy is quickly becoming a realistic treatment option for neurological disorders. Advancements in global central nervous system (CNS) gene delivery, in particular, have accelerated to the point that treatments for neurological disorders such as lysosomal storage diseases seem within reach. Other neurodevelopmental disorders, such as Rett Syndrome, Fragile X, and autism still face significant obstacles to overcome before a viable human gene therapy can be considered. This review focuses on the most common CNS gene delivery vehicle, adeno-associated virus (AAV), and the current state of AAV vector design and delivery for CNS gene therapy. Relevant examples of gene therapy studies for neurodevelopmental disorders, as well as outstanding challenges, are discussed. This article is part of the Special Issue entitled 'Neurodevelopmental Disorders'.
随着最近一些临床成功案例的出现，基因治疗正迅速成为治疗神经紊乱的一种可行的治疗选择。特别是全球中枢神经系统（CNS）基因传递方面的进步，已经迅速发展到治疗神经紊乱的方法，如溶酶体储存疾病似乎已经触手可及。其他神经发育障碍，如雷特综合征、脆性 X 综合征和自闭症，在可行的人类基因治疗被考虑之前，仍然面临着重大障碍需要克服。这篇综述重点介绍了最常见的中枢神经系统基因传递载体，腺相关病毒（AAV），以及目前中枢神经系统基因治疗的 AAV 载体设计和传递的情况。讨论了神经发育障碍基因治疗研究的相关实例，以及突出的挑战。本文是特刊“神经发育障碍”的一部分。
