Gene therapy for Parkinson's disease.
Identifieur interne : 001934 ( PubMed/Curation ); précédent : 001933; suivant : 001935Gene therapy for Parkinson's disease.
Auteurs : Tomas Bjorklund [Suède] ; Jeffrey H. KordowerSource :
- Movement disorders : official journal of the Movement Disorder Society [ 1531-8257 ] ; 2010.
English descriptors
- KwdEn :
- Animals, Clinical Trials, Phase II as Topic, Dopamine (administration & dosage), Enzymes (metabolism), Enzymes (therapeutic use), Genetic Therapy (methods), Glutamic Acid (metabolism), Humans, Intercellular Signaling Peptides and Proteins (genetics), Intercellular Signaling Peptides and Proteins (metabolism), Parkinson Disease (genetics), Parkinson Disease (metabolism), Parkinson Disease (therapy).
- MESH :
- chemical , administration & dosage : Dopamine.
- chemical , genetics : Intercellular Signaling Peptides and Proteins.
- chemical , metabolism : Enzymes, Glutamic Acid, Intercellular Signaling Peptides and Proteins.
- chemical , therapeutic use : Enzymes.
- genetics : Parkinson Disease.
- metabolism : Parkinson Disease.
- methods : Genetic Therapy.
- therapy : Parkinson Disease.
- Animals, Clinical Trials, Phase II as Topic, Humans.
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.
DOI: 10.1002/mds.22785
PubMed: 20187249
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