✦ LIBER ✦

Gene therapy for Parkinson's disease

✍ Scribed by Tomas Bjorklund; Jeffrey H. Kordower

Publisher: John Wiley and Sons
Year: 2010
Tongue: English
Weight: 133 KB
Volume: 25
Category: Article
ISSN: 0885-3185
DOI: 10.1002/mds.22785

No coin nor oath required. For personal study only.

✦ Synopsis

Abstract

The once fantastic theoretical concept that patients with Parkinson's disease (PD) would receive gene therapy in an attempt to alleviate their symptoms and potentially modify the course of their disease has become a reality. On the basis of positive preclinical data, four different gene therapy approaches are currently in Phase I or Phase II clinical trials. Some approaches are intended to increase levels of endogenous dopamine or enhance the function of the prodrug levodopa. Others are intended to normalize basal ganglia circuitry by reducing the PD‐related overactivity of specific brain structures such as the subthalamic nucleus. Each is intended for symptomatic benefit. Finally, gene delivery of trophic factors that not only augment dopaminergic function but are potentially disease modifying has a strong preclinical database and are also in clinical trials. Each of these approaches is discussed in the present review. © 2010 Movement Disorder Society

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