Gene therapy for dopamine replacement.
(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.
Please use this url to cite or link to this publication:
https://lup.lub.lu.se/record/1711470
- author
- Björklund, Tomas LU ; Cederfjäll, Erik LU and Kirik, Deniz LU
- organization
- publishing date
- 2010
- 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
- 2016-04-04 09:09:36
- date last changed
- 2022-03-15 17:59:17
@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}},
doi = {{10.1016/S0079-6123(10)84012-9}},
volume = {{184}},
year = {{2010}},
}