Gene therapy for Parkinson's disease.
(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)
Please use this url to cite or link to this publication:
https://lup.lub.lu.se/record/1582808
- author
- Björklund, Tomas LU and Kordower, Jeffrey H
- organization
- publishing date
- 2010
- type
- Contribution to journal
- publication status
- published
- subject
- in
- Movement Disorders
- volume
- 25 Suppl 1
- pages
- 161 - 173
- publisher
- John Wiley & Sons Inc.
- external identifiers
-
- wos:000276137000030
- pmid:20187249
- scopus:77953422395
- pmid:20187249
- 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
- 2016-04-04 07:20:12
- date last changed
- 2022-05-16 19:58:19
@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 Inc.}}, series = {{Movement Disorders}}, title = {{Gene therapy for Parkinson's disease.}}, url = {{http://dx.doi.org/10.1002/mds.22785}}, doi = {{10.1002/mds.22785}}, volume = {{25 Suppl 1}}, year = {{2010}}, }