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Gene therapy for dopamine replacement.

Björklund, Tomas LU ; Cederfjäll, Erik LU and Kirik, Deniz LU (2010) In Progress in Brain Research 184. p.221-235
Abstract
Dopamine replacement for Parkinson's disease (PD) have seen three major iterations of improvements since the introduction of l-3,4-dihydroxyphenylalanine (l-DOPA) pharmacotherapy: dopamine receptor agonists, ex vivo gene transfer for cell transplantation and most recently in vivo gene therapy. In this chapter, we describe the principles behind viral vector-mediated enzyme replacement in PD. We focus on the enzymes involved in the dopamine synthesis and their internal regulation, the early experimental work on gene therapy using different viral vector types and selection of transgenes, and finally discuss the recently completed early phase clinical trials in PD patients.
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Progress in Brain Research
volume
184
pages
221 - 235
publisher
Elsevier
external identifiers
  • wos:000287856300013
  • pmid:20887878
  • scopus:77957224858
ISSN
1875-7855
DOI
10.1016/S0079-6123(10)84012-9
language
English
LU publication?
yes
id
d16ece40-41da-4e32-bd73-9f0d0dc50139 (old id 1711470)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/20887878?dopt=Abstract
date added to LUP
2010-11-05 12:23:50
date last changed
2017-03-03 16:30:28
@article{d16ece40-41da-4e32-bd73-9f0d0dc50139,
  abstract     = {Dopamine replacement for Parkinson's disease (PD) have seen three major iterations of improvements since the introduction of l-3,4-dihydroxyphenylalanine (l-DOPA) pharmacotherapy: dopamine receptor agonists, ex vivo gene transfer for cell transplantation and most recently in vivo gene therapy. In this chapter, we describe the principles behind viral vector-mediated enzyme replacement in PD. We focus on the enzymes involved in the dopamine synthesis and their internal regulation, the early experimental work on gene therapy using different viral vector types and selection of transgenes, and finally discuss the recently completed early phase clinical trials in PD patients.},
  author       = {Björklund, Tomas and Cederfjäll, Erik and Kirik, Deniz},
  issn         = {1875-7855},
  language     = {eng},
  pages        = {221--235},
  publisher    = {Elsevier},
  series       = {Progress in Brain Research},
  title        = {Gene therapy for dopamine replacement.},
  url          = {http://dx.doi.org/10.1016/S0079-6123(10)84012-9},
  volume       = {184},
  year         = {2010},
}