Current status of gene therapy trials for Parkinson's disease

被引:41
作者
Fiandaca, Massimo [1 ,2 ]
Forsayeth, John [1 ]
Banklewiez, Krystof [1 ]
机构
[1] Univ Calif San Francisco, Dept Neurol Surg, Movement Disorder Res Program, San Francisco, CA 94103 USA
[2] LifeBridge Hlth Brain & Spine Inst, Dept Neurosurg, Baltimore, MD 21209 USA
关键词
Parkinson's disease; gene therapy; clinical trials; viral vectors; CERE-120; neurturin; GDNF; AAV2-hAADC; AAV-GAD; striatum; subthalamic nucleus; convection-enhanced delivery;
D O I
10.1016/j.expneurol.2007.08.009
中图分类号
Q189 [神经科学];
学科分类号
071006 ;
摘要
The incidence of Parkinson's disease (PD) increases greatly with age, and the baby-boomer population can expect to generate a large number of individuals with the disease, all of whom will have significantly increased medical care needs over periods of 20 years or more. This emerging healthcare burden to our society calls for accelerated efforts to understand this disease better and treat it more effectively. The growing interest in gene therapy grew out of a recognition that new medicines may be needed to combat the relentless progression of the disease in the face of conventional pharmaco-therapies and surgical interventions that have so far failed to offer more than palliative relief The potential of gene therapy to alter dramatically the course of the disease lies very much with the challenge of converting a research tool into a medical option, a process that clearly requires a unique combination of rigor and flexibility. In this review, we examine the unique aspects of gene therapy that make its use in PD attractive, but also analyze the difficulties of employing a medicine that acts for the rest of the patient's life. (c) 2007 Published by Elsevier Inc.
引用
收藏
页码:51 / 57
页数:7
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