Gene Therapy for Parkinson’s Disease
Elgstrand, Erika LU ; Quintino, Luis LU
; Manfre, Giuseppe
LU
and Lundberg, Cecilia
LU
(2014)
82.
p.181-191
- Abstract
- Gene therapy is a promising future tool for treatment of Parkinson’s disease (PD) and several different strategies are currently being evaluated. Although many of these strategies have shown promising results in animal models of PD and parkinsonian patients, some have been less effective and caused adverse side effects. A vector system with high specificity and appropriate expression level of the transgene is needed to make gene therapy for PD as safe and beneficial as possible. The vector should also be relevant for the disease. Here, we present a method to design promoters relevant for PD, using microarray data from patients, and validation of these in vivo. The method also includes fine-tuning of promoter candidates by adding miRNA... (More)
- Gene therapy is a promising future tool for treatment of Parkinson’s disease (PD) and several different strategies are currently being evaluated. Although many of these strategies have shown promising results in animal models of PD and parkinsonian patients, some have been less effective and caused adverse side effects. A vector system with high specificity and appropriate expression level of the transgene is needed to make gene therapy for PD as safe and beneficial as possible. The vector should also be relevant for the disease. Here, we present a method to design promoters relevant for PD, using microarray data from patients, and validation of these in vivo. The method also includes fine-tuning of promoter candidates by adding miRNA target sites to increase cell specificity. (Less)
Please use this url to cite or link to this publication:
https://lup.lub.lu.se/record/cd93753a-b0d9-49ee-84ee-2ea607108818
- author
- Elgstrand, Erika
LU
; Quintino, Luis
LU
; Manfre, Giuseppe
LU
and Lundberg, Cecilia
LU
- organization
- publishing date
- 2014
- type
- Chapter in Book/Report/Conference proceeding
- publication status
- published
- subject
- host publication
- Viral Vector Approaches in Neurobiology and Brain Diseases
- editor
- Brambilla, Riccardo
- volume
- 82
- article number
- 10
- pages
- 11 pages
- publisher
- Springer
- external identifiers
-
- scopus:84927614000
- ISBN
- 9781627036092
- 978-1-62703-610-8
- DOI
- 10.1007/978-1-62703-610-8_10
- language
- English
- LU publication?
- yes
- id
- cd93753a-b0d9-49ee-84ee-2ea607108818
- date added to LUP
- 2017-04-05 13:26:00
- date last changed
- 2024-01-13 18:16:59
@inbook{cd93753a-b0d9-49ee-84ee-2ea607108818,
abstract = {{Gene therapy is a promising future tool for treatment of Parkinson’s disease (PD) and several different strategies are currently being evaluated. Although many of these strategies have shown promising results in animal models of PD and parkinsonian patients, some have been less effective and caused adverse side effects. A vector system with high specificity and appropriate expression level of the transgene is needed to make gene therapy for PD as safe and beneficial as possible. The vector should also be relevant for the disease. Here, we present a method to design promoters relevant for PD, using microarray data from patients, and validation of these in vivo. The method also includes fine-tuning of promoter candidates by adding miRNA target sites to increase cell specificity.}},
author = {{Elgstrand, Erika and Quintino, Luis and Manfre, Giuseppe and Lundberg, Cecilia}},
booktitle = {{Viral Vector Approaches in Neurobiology and Brain Diseases}},
editor = {{Brambilla, Riccardo}},
isbn = {{9781627036092}},
language = {{eng}},
pages = {{181--191}},
publisher = {{Springer}},
title = {{Gene Therapy for Parkinson’s Disease}},
url = {{http://dx.doi.org/10.1007/978-1-62703-610-8_10}},
doi = {{10.1007/978-1-62703-610-8_10}},
volume = {{82}},
year = {{2014}},
}