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Scientific rationale for the development of gene therapy strategies for Parkinson's disease.

Björklund, Tomas LU and Kirik, Deniz LU (2009) In Biochimica et Biophysica Acta - Molecular Basis of Disease 1792(7). p.703-713
Abstract
The ever-evolving understanding of the neuronal systems involved in Parkinson's disease together with the recent advances in recombinant viral vector technology has led to the development of several gene therapy applications that are now entering into clinical testing phase. To date, four fundamentally different approaches have been pursued utilizing recombinant adeno-associated virus and lentiviruses as vectors for delivery. These strategies aim either to restore the lost brain functions by substitution of enzymes critical for synthesis of neurotransmitters or neurotrophic factors as a means to boost the function of remaining neurons in the diseased brain. In this review we discuss the differences in mechanism of action and describe the... (More)
The ever-evolving understanding of the neuronal systems involved in Parkinson's disease together with the recent advances in recombinant viral vector technology has led to the development of several gene therapy applications that are now entering into clinical testing phase. To date, four fundamentally different approaches have been pursued utilizing recombinant adeno-associated virus and lentiviruses as vectors for delivery. These strategies aim either to restore the lost brain functions by substitution of enzymes critical for synthesis of neurotransmitters or neurotrophic factors as a means to boost the function of remaining neurons in the diseased brain. In this review we discuss the differences in mechanism of action and describe the scientific rationale behind the currently tested gene therapy approaches for Parkinson's disease in some detail and pinpoint their individual unique strengths and weaknesses. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Biochimica et Biophysica Acta - Molecular Basis of Disease
volume
1792
issue
7
pages
703 - 713
publisher
Elsevier
external identifiers
  • wos:000267960200013
  • pmid:19254760
  • scopus:68649084201
ISSN
0925-4439
DOI
10.1016/j.bbadis.2009.02.009
language
English
LU publication?
yes
id
e71ca5fe-f440-4c88-9bbe-9c9c78675721 (old id 1368091)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/19254760?dopt=Abstract
date added to LUP
2009-04-03 12:19:19
date last changed
2017-03-03 16:32:08
@article{e71ca5fe-f440-4c88-9bbe-9c9c78675721,
  abstract     = {The ever-evolving understanding of the neuronal systems involved in Parkinson's disease together with the recent advances in recombinant viral vector technology has led to the development of several gene therapy applications that are now entering into clinical testing phase. To date, four fundamentally different approaches have been pursued utilizing recombinant adeno-associated virus and lentiviruses as vectors for delivery. These strategies aim either to restore the lost brain functions by substitution of enzymes critical for synthesis of neurotransmitters or neurotrophic factors as a means to boost the function of remaining neurons in the diseased brain. In this review we discuss the differences in mechanism of action and describe the scientific rationale behind the currently tested gene therapy approaches for Parkinson's disease in some detail and pinpoint their individual unique strengths and weaknesses.},
  author       = {Björklund, Tomas and Kirik, Deniz},
  issn         = {0925-4439},
  language     = {eng},
  number       = {7},
  pages        = {703--713},
  publisher    = {Elsevier},
  series       = {Biochimica et Biophysica Acta - Molecular Basis of Disease},
  title        = {Scientific rationale for the development of gene therapy strategies for Parkinson's disease.},
  url          = {http://dx.doi.org/10.1016/j.bbadis.2009.02.009},
  volume       = {1792},
  year         = {2009},
}