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从帕金森病的移植治疗到基因治疗。

From transplants to gene therapy for Parkinson's disease.

作者信息

Freeman T B

机构信息

Division of Neurosurgery, University of South Florida, Tampa 33606, USA.

出版信息

Exp Neurol. 1997 Mar;144(1):47-50. doi: 10.1006/exnr.1996.6387.

DOI:10.1006/exnr.1996.6387
PMID:9126151
Abstract

The efficacy of fetal tissue transplants in the treatment of Parkinson's disease has been demonstrated preliminarily. However, results are short term, and have not been confirmed in prospective randomized controlled trials. Furthermore, although patients are improved, they remain severely disabled. There are several problems with the widespread clinical use of fetal tissue that make the use of molecular biological strategies more compelling. Several conceptual problems are shared by both fetal and molecular biological neuroreconstruction strategies. It is necessary to characterize the "dose" of the transplanted therapeutic agent as well as its volume of distribution. The symptoms that may improve following neuronal reconstruction are likely to be directly related to the somatotopic localization of the transplants; the duration of benefit should be long enough to be relevant in a chronic disease such as Parkinson's disease. Operative therapies that utilize gene therapy or other reconstructive neurosurgical techniques are likely to require novel clinical trial designs to demonstrate their efficacy.

摘要

胎儿组织移植治疗帕金森病的疗效已得到初步证实。然而,结果是短期的,尚未在前瞻性随机对照试验中得到证实。此外,尽管患者有所改善,但仍严重残疾。胎儿组织广泛临床应用存在若干问题,这使得分子生物学策略的应用更具吸引力。胎儿和分子生物学神经重建策略都存在几个概念性问题。有必要确定移植治疗剂的“剂量”及其分布体积。神经元重建后可能改善的症状可能与移植的躯体定位直接相关;受益持续时间应足够长,以对帕金森病等慢性疾病有意义。利用基因治疗或其他重建性神经外科技术的手术疗法可能需要新颖的临床试验设计来证明其疗效。

相似文献

1
From transplants to gene therapy for Parkinson's disease.从帕金森病的移植治疗到基因治疗。
Exp Neurol. 1997 Mar;144(1):47-50. doi: 10.1006/exnr.1996.6387.
2
Neural transplantation in Parkinson's disease.
Adv Neurol. 2001;86:435-45.
3
Subthalamic GAD gene transfer in Parkinson disease patients who are candidates for deep brain stimulation.对适合进行深部脑刺激的帕金森病患者进行丘脑底核谷氨酸脱羧酶基因转移。
Hum Gene Ther. 2001 Aug 10;12(12):1589-91.
4
Surgical treatment of Parkinson's disease.帕金森病的外科治疗
Am Fam Physician. 1996 Oct;54(5):1621-9.
5
[Cell therapy and other neuroregenerative strategies in Parkinson's disease (I)].
Rev Neurol. 2005;41(10):604-14.
6
[Biotherapies and Parkinson's disease].[生物疗法与帕金森病]
Rev Neurol (Paris). 2009 Nov;165(11):857-62. doi: 10.1016/j.neurol.2009.03.017. Epub 2009 May 31.
7
Treatment of Parkinson's disease. Symptomatic cell therapies: cells as biological minipumps.帕金森病的治疗。症状性细胞疗法:作为生物微型泵的细胞。
Eur Neurol. 1996;36(6):405-8.
8
Do patients with Parkinson's disease benefit from embryonic dopamine cell transplantation?帕金森病患者能从胚胎多巴胺细胞移植中获益吗?
J Neurol. 2003 Oct;250 Suppl 3:III44-6. doi: 10.1007/s00415-003-1308-5.
9
[Cell transplantation and gene therapy in Parkinson disease].[帕金森病的细胞移植与基因治疗]
Nord Med. 1996 Sep;111(7):225-9.
10
Parkinson's disease: new treatment strategies.帕金森病:新的治疗策略
Compr Ther. 1990 Dec;16(12):41-6.

引用本文的文献

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Lycium barbarum polysaccharide improves dopamine metabolism and symptoms in an MPTP-induced model of Parkinson's disease.枸杞多糖改善 MPTP 诱导的帕金森病模型中的多巴胺代谢和症状。
BMC Med. 2022 Oct 28;20(1):412. doi: 10.1186/s12916-022-02621-9.
2
Holistic Approach of Swiss Fetal Progenitor Cell Banking: Optimizing Safe and Sustainable Substrates for Regenerative Medicine and Biotechnology.瑞士胎儿祖细胞库的整体方法:为再生医学和生物技术优化安全且可持续的基质。
Front Bioeng Biotechnol. 2020 Oct 23;8:557758. doi: 10.3389/fbioe.2020.557758. eCollection 2020.
3
Induced pluripotent stem cells for modeling and cell therapy of Parkinson's disease.
用于帕金森病建模和细胞治疗的诱导多能干细胞。
Neural Regen Res. 2016 May;11(5):727-8. doi: 10.4103/1673-5374.182692.
4
Neurodegeneration and cell replacement.神经退行性变与细胞替代
Philos Trans R Soc Lond B Biol Sci. 2008 Jan 12;363(1489):153-70. doi: 10.1098/rstb.2006.2018.
5
Should we clone human beings? Cloning as a source of tissue for transplantation.我们应该克隆人类吗?克隆作为移植组织的来源。
J Med Ethics. 1999 Apr;25(2):87-95. doi: 10.1136/jme.25.2.87.