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)
Please use this url to cite or link to this publication:
https://lup.lub.lu.se/record/1368091
- author
- Björklund, Tomas
LU
and Kirik, Deniz
LU
- organization
- publishing date
- 2009
- 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
- 2016-04-04 09:22:46
- date last changed
- 2025-02-17 13:15:29
@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 = {{https://lup.lub.lu.se/search/files/5308343/1390922.pdf}},
doi = {{10.1016/j.bbadis.2009.02.009}},
volume = {{1792}},
year = {{2009}},
}