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Gene Therapy for Parkinson’s Disease

Elgstrand, Erika LU ; Quintino, Luis LU ; Manfre, Giuseppe LU and Lundberg, Cecilia LU (2014) In Viral Vector Approaches in Neurobiology and Brain Diseases 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)
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author
organization
publishing date
type
Chapter in Book/Report/Conference proceeding
publication status
published
subject
in
Viral Vector Approaches in Neurobiology and Brain Diseases
editor
Brambilla, Riccardo and
volume
82
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
2017-04-09 04:56:18
@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},
  editor       = {Brambilla, Riccardo},
  isbn         = {9781627036092},
  language     = {eng},
  pages        = {181--191},
  publisher    = {Springer},
  series       = {Viral Vector Approaches in Neurobiology and Brain Diseases },
  title        = {Gene Therapy for Parkinson’s Disease},
  url          = {http://dx.doi.org/10.1007/978-1-62703-610-8_10},
  volume       = {82},
  year         = {2014},
}