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Gene therapy for dopamine replacement in Parkinson's disease.

Björklund, Anders LU orcid ; Björklund, Tomas LU and Kirik, Deniz LU (2009) In Science Translational Medicine 1(2). p.2-2
Abstract
The introduction of L-dopa (L-3,4-dihydroxyphenylalanine) therapy 40 years ago was a revolution in the treatment of patients with Parkinson s disease (PD). With time, however, the shortcomings of oral L-dopa medication became apparent, in particular the appearance of troublesome side effects, expressed as involuntary movements (dyskinesias) that developed over time in many patients. A gene therapy approach, aimed at restoring dopamine synthesis in the affected brain by viral vector delivery of genes that encode the dopamine-synthesizing enzymes, may offer a solution to this problem. Now, a team of French and UK researchers reports promising results in a nonhuman primate model of PD, paving the way for clinical trials of this... (More)
The introduction of L-dopa (L-3,4-dihydroxyphenylalanine) therapy 40 years ago was a revolution in the treatment of patients with Parkinson s disease (PD). With time, however, the shortcomings of oral L-dopa medication became apparent, in particular the appearance of troublesome side effects, expressed as involuntary movements (dyskinesias) that developed over time in many patients. A gene therapy approach, aimed at restoring dopamine synthesis in the affected brain by viral vector delivery of genes that encode the dopamine-synthesizing enzymes, may offer a solution to this problem. Now, a team of French and UK researchers reports promising results in a nonhuman primate model of PD, paving the way for clinical trials of this enzyme-replacement approach. (Less)
Please use this url to cite or link to this publication:
author
; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Science Translational Medicine
volume
1
issue
2
pages
2 - 2
publisher
American Association for the Advancement of Science (AAAS)
external identifiers
  • wos:000277147600002
  • pmid:20368161
  • scopus:76949092638
  • pmid:20368161
ISSN
1946-6242
DOI
10.1126/scitranslmed.3000350
language
English
LU publication?
yes
id
56c94388-dc90-4bab-944b-60887490136c (old id 1595589)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/20368161?dopt=Abstract
date added to LUP
2016-04-04 07:26:46
date last changed
2022-02-13 05:00:36
@article{56c94388-dc90-4bab-944b-60887490136c,
  abstract     = {{The introduction of L-dopa (L-3,4-dihydroxyphenylalanine) therapy 40 years ago was a revolution in the treatment of patients with Parkinson s disease (PD). With time, however, the shortcomings of oral L-dopa medication became apparent, in particular the appearance of troublesome side effects, expressed as involuntary movements (dyskinesias) that developed over time in many patients. A gene therapy approach, aimed at restoring dopamine synthesis in the affected brain by viral vector delivery of genes that encode the dopamine-synthesizing enzymes, may offer a solution to this problem. Now, a team of French and UK researchers reports promising results in a nonhuman primate model of PD, paving the way for clinical trials of this enzyme-replacement approach.}},
  author       = {{Björklund, Anders and Björklund, Tomas and Kirik, Deniz}},
  issn         = {{1946-6242}},
  language     = {{eng}},
  number       = {{2}},
  pages        = {{2--2}},
  publisher    = {{American Association for the Advancement of Science (AAAS)}},
  series       = {{Science Translational Medicine}},
  title        = {{Gene therapy for dopamine replacement in Parkinson's disease.}},
  url          = {{http://dx.doi.org/10.1126/scitranslmed.3000350}},
  doi          = {{10.1126/scitranslmed.3000350}},
  volume       = {{1}},
  year         = {{2009}},
}