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Gene therapy for Parkinson's disease.

Björklund, Tomas LU and Kordower, Jeffrey H (2010) In Movement Disorders 25 Suppl 1. p.161-173
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... (More)
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. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Movement Disorders
volume
25 Suppl 1
pages
161 - 173
publisher
John Wiley & Sons
external identifiers
  • wos:000276137000030
  • pmid:20187249
  • scopus:77953422395
ISSN
0885-3185
DOI
10.1002/mds.22785
language
English
LU publication?
yes
id
4934e035-d3e5-4512-925b-dd9c286b5209 (old id 1582808)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/20187249?dopt=Abstract
date added to LUP
2010-04-07 09:34:26
date last changed
2018-05-29 09:28:30
@article{4934e035-d3e5-4512-925b-dd9c286b5209,
  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.},
  author       = {Björklund, Tomas and Kordower, Jeffrey H},
  issn         = {0885-3185},
  language     = {eng},
  pages        = {161--173},
  publisher    = {John Wiley & Sons},
  series       = {Movement Disorders},
  title        = {Gene therapy for Parkinson's disease.},
  url          = {http://dx.doi.org/10.1002/mds.22785},
  volume       = {25 Suppl 1},
  year         = {2010},
}