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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.

DOI:10.3233/JPD-212710
PMID:34366372
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8543258/
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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1
Improved Delivery Methods for Gene Therapy and Cell Transplantation in Parkinson's Disease.帕金森病基因治疗和细胞移植的改良递送方法。
J Parkinsons Dis. 2021;11(s2):S199-S206. doi: 10.3233/JPD-212710.
2
Focused ultrasound opening of the blood-brain barrier for treatment of Parkinson's disease.聚焦超声打开血脑屏障治疗帕金森病。
Mov Disord. 2019 Sep;34(9):1274-1278. doi: 10.1002/mds.27722. Epub 2019 May 28.
3
Blood-brain barrier opening with focused ultrasound in experimental models of Parkinson's disease.在帕金森病的实验模型中,利用聚焦超声打开血脑屏障。
Mov Disord. 2019 Sep;34(9):1252-1261. doi: 10.1002/mds.27804. Epub 2019 Jul 30.
4
Future and current surgical therapies in Parkinson's disease.帕金森病的未来及当前手术治疗方法
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Genetically engineered mesenchymal stem cells with dopamine synthesis for Parkinson's disease in animal models.用于动物模型帕金森病的具有多巴胺合成能力的基因工程间充质干细胞。
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本文引用的文献

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Gene Therapy in Movement Disorders: A Systematic Review of Ongoing and Completed Clinical Trials.运动障碍的基因治疗:对正在进行和已完成临床试验的系统评价
Front Neurol. 2021 Apr 6;12:648532. doi: 10.3389/fneur.2021.648532. eCollection 2021.
2
Therapeutic Agent Delivery Across the Blood-Brain Barrier Using Focused Ultrasound.利用聚焦超声实现血脑屏障内治疗药物的递送。
Annu Rev Biomed Eng. 2021 Jul 13;23:89-113. doi: 10.1146/annurev-bioeng-062117-121238. Epub 2021 Mar 22.
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Convection-enhanced drug delivery for glioblastoma: a review.增强型局部化疗治疗脑胶质母细胞瘤:综述。
J Neurooncol. 2021 Feb;151(3):415-427. doi: 10.1007/s11060-020-03408-9. Epub 2021 Feb 21.
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Use of a novel ball-joint guide array for magnetic resonance imaging-guided cannula placement and convective delivery: technical note.一种用于磁共振成像引导下套管置入和对流给药的新型球窝导向阵列的应用:技术说明。
J Neurosurg. 2020 Oct 23;135(2):651-657. doi: 10.3171/2020.6.JNS201564. Print 2021 Aug 1.
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Anti-α-synuclein ASO delivered to monoamine neurons prevents α-synuclein accumulation in a Parkinson's disease-like mouse model and in monkeys.抗 α-突触核蛋白 ASO 递送至单胺能神经元可预防帕金森病样小鼠模型和猴子中 α-突触核蛋白的累积。
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Invest Radiol. 2019 Aug;54(8):453-463. doi: 10.1097/RLI.0000000000000567.
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Trial of magnetic resonance-guided putaminal gene therapy for advanced Parkinson's disease.磁共振引导壳核基因治疗晚期帕金森病的试验。
Mov Disord. 2019 Jul;34(7):1073-1078. doi: 10.1002/mds.27724. Epub 2019 May 30.
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Effects of surgical targeting in laser interstitial thermal therapy for mesial temporal lobe epilepsy: A multicenter study of 234 patients.手术靶向在激光间质热疗治疗内侧颞叶癫痫中的作用:234 例多中心研究。
Epilepsia. 2019 Jun;60(6):1171-1183. doi: 10.1111/epi.15565. Epub 2019 May 21.
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Brain. 2019 Mar 1;142(3):512-525. doi: 10.1093/brain/awz023.