帕金森病的营养因子治疗
Trophic factors therapy in Parkinson's disease.
作者信息
Ramaswamy Shilpa, Soderstrom Katherine E, Kordower Jeffrey H
机构信息
Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, USA.
出版信息
Prog Brain Res. 2009;175:201-16. doi: 10.1016/S0079-6123(09)17514-3.
Abstract
Parkinson's disease (PD) is a progressive, neurodegenerative disorder for which there is currently no effective neuroprotective therapy. Patients are typically treated with a combination of drug therapies and/or receive deep brain stimulation to combat behavioral symptoms. The ideal candidate therapy would be the one which prevents neurodegeneration in the brain, thereby halting the progression of debilitating disease symptoms. Neurotrophic factors have been in the forefront of PD research, and clinical trials have been initiated using members of the GDNF family of ligands (GFLs). GFLs have been shown to be trophic to ventral mesencephalic cells, thereby making them good candidates for PD research. This paper examines the use of GDNF and neurturin, two members of the GFL, in both animal models of PD and clinical trials.
摘要
帕金森病（PD）是一种进行性神经退行性疾病，目前尚无有效的神经保护疗法。患者通常采用药物疗法联合治疗和/或接受深部脑刺激来对抗行为症状。理想的候选疗法应该是能够预防大脑神经退行性变，从而阻止使人衰弱的疾病症状进展的疗法。神经营养因子一直处于帕金森病研究的前沿，目前已经启动了使用胶质细胞源性神经营养因子（GDNF）配体家族（GFLs）成员的临床试验。GFLs已被证明对腹侧中脑细胞具有营养作用，因此使其成为帕金森病研究的良好候选对象。本文研究了GFLs的两个成员GDNF和神经营养素在帕金森病动物模型和临床试验中的应用。
相似文献
1
Trophic factors therapy in Parkinson's disease.帕金森病的营养因子治疗
Prog Brain Res. 2009;175:201-16. doi: 10.1016/S0079-6123(09)17514-3.
2
Neurotrophic factor therapy for Parkinson's disease.神经营养因子治疗帕金森病。
Prog Brain Res. 2010;184:237-64. doi: 10.1016/S0079-6123(10)84013-0.
3
Prospects of Neurotrophic Factors for Parkinson's Disease: Comparison of Protein and Gene Therapy.帕金森病的神经营养因子前景：蛋白质疗法与基因疗法的比较
Hum Gene Ther. 2015 Aug;26(8):550-9. doi: 10.1089/hum.2015.065.
4
Trophic factor gene therapy for Parkinson's disease.神经营养因子基因治疗帕金森病。
Mov Disord. 2013 Jan;28(1):96-109. doi: 10.1002/mds.25344.
5
Treatment of Parkinson's disease with trophic factors.用营养因子治疗帕金森病。
Neurotherapeutics. 2008 Apr;5(2):270-80. doi: 10.1016/j.nurt.2008.02.003.
6
Current disease modifying approaches to treat Parkinson's disease.目前用于治疗帕金森病的疾病修饰方法。
Cell Mol Life Sci. 2016 Apr;73(7):1365-79. doi: 10.1007/s00018-015-2101-1. Epub 2015 Nov 30.
7
Gene transfer techniques for the delivery of GDNF in Parkinson's disease.用于帕金森病中递送胶质细胞源性神经营养因子（GDNF）的基因转移技术。
Novartis Found Symp. 2000;231:202-15; discussion 215-9, 302-6. doi: 10.1002/0470870834.ch13.
8
AAV2-Neurturin for Parkinson's Disease: What Lessons Have We Learned?用于帕金森病的腺相关病毒2型神经营养因子：我们学到了什么？
Methods Mol Biol. 2016;1382:485-90. doi: 10.1007/978-1-4939-3271-9_32.
9
GDNF family ligands: a potential future for Parkinson's disease therapy.胶质细胞源性神经营养因子家族配体：帕金森病治疗的潜在未来。
CNS Neurol Disord Drug Targets. 2011 Sep 1;10(6):703-11. doi: 10.2174/187152711797247876.
10
GDNF and Parkinson's Disease: Where Next? A Summary from a Recent Workshop.GDNF 与帕金森病：未来之路？近期研讨会总结。
J Parkinsons Dis. 2020;10(3):875-891. doi: 10.3233/JPD-202004.
引用本文的文献
1
Cerebrospinal Fluid EV Concentration and Size Are Altered in Alzheimer's Disease and Dementia with Lewy Bodies.脑脊髓液外泌体浓度和大小在阿尔茨海默病和路易体痴呆中发生改变。
Cells. 2022 Jan 28;11(3):462. doi: 10.3390/cells11030462.
2
Plasma Extracellular Vesicle Size and Concentration Are Altered in Alzheimer's Disease, Dementia With Lewy Bodies, and Frontotemporal Dementia.阿尔茨海默病、路易体痴呆和额颞叶痴呆患者的血浆细胞外囊泡大小和浓度发生改变。
Front Cell Dev Biol. 2021 May 11;9:667369. doi: 10.3389/fcell.2021.667369. eCollection 2021.
3
GDNF-expressing macrophages restore motor functions at a severe late-stage, and produce long-term neuroprotective effects at an early-stage of Parkinson's disease in transgenic Parkin Q311X(A) mice.GDNF 表达的巨噬细胞可在帕金森病转基因 Parkin Q311X(A) 小鼠的严重晚期恢复运动功能，并在早期产生长期的神经保护作用。
J Control Release. 2019 Dec 10;315:139-149. doi: 10.1016/j.jconrel.2019.10.027. Epub 2019 Oct 31.
4
Secretome of Undifferentiated Neural Progenitor Cells Induces Histological and Motor Improvements in a Rat Model of Parkinson's Disease.未分化神经祖细胞的分泌组可诱导帕金森病大鼠模型的组织学和运动改善。
Stem Cells Transl Med. 2018 Nov;7(11):829-838. doi: 10.1002/sctm.18-0009. Epub 2018 Sep 20.
5
Impact of the Secretome of Human Mesenchymal Stem Cells on Brain Structure and Animal Behavior in a Rat Model of Parkinson's Disease.人骨髓间充质干细胞分泌组对帕金森病大鼠模型脑结构和动物行为的影响。
Stem Cells Transl Med. 2017 Feb;6(2):634-646. doi: 10.5966/sctm.2016-0071. Epub 2016 Sep 22.
6
Functional Diversity of Neurotrophin Actions on the Oculomotor System.神经营养因子对动眼系统作用的功能多样性
Int J Mol Sci. 2016 Dec 1;17(12):2016. doi: 10.3390/ijms17122016.
7
Neuropharmacologic Approaches to Restore the Brain's Microenvironment.恢复大脑微环境的神经药理学方法。
J Neuroimmune Pharmacol. 2016 Sep;11(3):484-94. doi: 10.1007/s11481-016-9686-5. Epub 2016 Jun 28.
8
Comparative Analysis of the Effects of Neurotrophic Factors CDNF and GDNF in a Nonhuman Primate Model of Parkinson's Disease.神经营养因子CDNF和GDNF在帕金森病非人灵长类动物模型中的作用比较分析
PLoS One. 2016 Feb 22;11(2):e0149776. doi: 10.1371/journal.pone.0149776. eCollection 2016.
9
Noninvasive, Targeted, and Non-Viral Ultrasound-Mediated GDNF-Plasmid Delivery for Treatment of Parkinson's Disease.用于治疗帕金森病的非侵入性、靶向性和非病毒超声介导的胶质细胞源性神经营养因子质粒递送
Sci Rep. 2016 Jan 20;6:19579. doi: 10.1038/srep19579.
10
TRPV1 on astrocytes rescues nigral dopamine neurons in Parkinson's disease via CNTF.星形胶质细胞上的TRPV1通过睫状神经营养因子拯救帕金森病中的黑质多巴胺能神经元。
Brain. 2015 Dec;138(Pt 12):3610-22. doi: 10.1093/brain/awv297. Epub 2015 Oct 21.
本文引用的文献
1
Absolute requirement of GDNF for adult catecholaminergic neuron survival.胶质细胞源性神经营养因子对成年儿茶酚胺能神经元存活的绝对需求。
Nat Neurosci. 2008 Jul;11(7):755-61. doi: 10.1038/nn.2136. Epub 2008 Jun 8.
2
Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial.向特发性帕金森病患者脑内注射CERE-120（腺相关病毒2型-神经营养因子）的安全性和耐受性：一项开放标签的I期试验。
Lancet Neurol. 2008 May;7(5):400-8. doi: 10.1016/S1474-4422(08)70065-6. Epub 2008 Apr 2.
3
AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: long-term efficacy and tolerability of CERE-120 for Parkinson's disease.腺相关病毒2介导的人神经营养因子向大鼠黑质纹状体系统的递送：CERE-120治疗帕金森病的长期疗效和耐受性
Neurobiol Dis. 2007 Jul;27(1):67-76. doi: 10.1016/j.nbd.2007.04.003. Epub 2007 Apr 19.
4
Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys.向纹状体递送CERE-120（一种编码人神经营养素的腺相关病毒2型载体）可增强老年猴多巴胺能黑质纹状体系统的活性。
Mov Disord. 2007 Jun 15;22(8):1124-32. doi: 10.1002/mds.21503.
5
Unilateral intraputamenal glial cell line-derived neurotrophic factor in patients with Parkinson disease: response to 1 year of treatment and 1 year of withdrawal.帕金森病患者单侧壳核内注射胶质细胞源性神经营养因子：1年治疗及1年撤药后的反应
J Neurosurg. 2007 Apr;106(4):614-20. doi: 10.3171/jns.2007.106.4.614.
6
Transplantation of bone marrow stromal cells containing the neurturin gene in rat model of Parkinson's disease.在帕金森病大鼠模型中移植含神经营养因子基因的骨髓基质细胞
Brain Res. 2007 Apr 20;1142:206-16. doi: 10.1016/j.brainres.2006.12.061. Epub 2006 Dec 24.
7
Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys.通过AAV2（CERE-120）介导的基因转移递送神经营养因子在MPTP处理的猴子中提供结构和功能上的神经保护及神经修复。
Ann Neurol. 2006 Dec;60(6):706-15. doi: 10.1002/ana.21032.
8
Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's disease.通过CERE-120（一种用于治疗帕金森病中多巴胺能神经元变性的腺相关病毒2型载体）将神经营养素递送至纹状体。
Mol Ther. 2007 Jan;15(1):62-8. doi: 10.1038/sj.mt.6300010.
9
Dopaminergic neuroprotection by neurturin-expressing c17.2 neural stem cells in a rat model of Parkinson's disease.在帕金森病大鼠模型中，表达神经营养因子的C17.2神经干细胞对多巴胺能神经元的神经保护作用。
Parkinsonism Relat Disord. 2007 Mar;13(2):77-88. doi: 10.1016/j.parkreldis.2006.07.015. Epub 2006 Sep 11.
10
Point source concentration of GDNF may explain failure of phase II clinical trial.胶质细胞源性神经营养因子的点源浓度可能解释了II期临床试验的失败。
Exp Neurol. 2006 Dec;202(2):497-505. doi: 10.1016/j.expneurol.2006.07.015. Epub 2006 Sep 7.
Zotero 插件