帕金森病基因治疗和细胞移植的改良递送方法。
Improved Delivery Methods for Gene Therapy and Cell Transplantation in Parkinson's Disease.
机构信息
Department of Neurosurgery, University of Arizona, Tucson, AZ, USA.
出版信息
J Parkinsons Dis. 2021;11(s2):S199-S206. doi: 10.3233/JPD-212710.
Abstract
A number of cell transplantation and gene therapy trials have been performed over the last three decades in an effort to restore function in Parkinson's disease. Much has been learned about optimizing delivery methods for these therapeutics. This is particularly true in gene therapy, which has predominated the clinical trial landscape in recent years; however, cell transplantation for Parkinson's disease is currently undergoing a renaissance. Innovations such as cannula design, iMRI-guided surgery and an evolution in delivery strategy has radically changed the way investigators approach clinical trial design. Future therapeutic strategies may employ newer delivery methods such as chronically implanted infusion devices and focal opening of the blood brain barrier with focused ultrasound.
摘要
在过去的三十年中，已经进行了许多细胞移植和基因治疗试验，以努力恢复帕金森病的功能。在优化这些治疗方法的传递方法方面已经学到了很多。近年来，这在基因治疗中尤其如此，基因治疗在临床试验领域占据主导地位；然而，帕金森病的细胞移植目前正在复兴。创新，如套管设计、iMRI 引导手术和传递策略的演变，彻底改变了研究人员进行临床试验设计的方式。未来的治疗策略可能采用更新的传递方法，例如慢性植入式输注装置和聚焦超声打开血脑屏障的焦点。
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