Advanced

Modeling CNS neurodegeneration by overexpression of disease-causing proteins using viral vectors.

Kirik, Deniz LU and Björklund, Anders LU (2003) In Trends in Neurosciences 26(7). p.386-392
Abstract
Defective handling of proteins is a central feature of major neurodegenerative diseases. The discovery that neuronal dysfunction or degeneration can be caused by mutations in single cellular proteins has given new opportunities to model the underlying disease processes by genetic modification of cells in vitro or by generation of transgenic animals carrying the disease-causing gene. Recent developments in recombinant viral-vector technology have opened up an interesting alternative possibility, based on direct gene transfer to selected subregions or subsets of neurons in the brain. Using the highly efficient adeno-associated virus or lentivirus vectors, recent reports have shown that overexpression of mutated human huntingtin or... (More)
Defective handling of proteins is a central feature of major neurodegenerative diseases. The discovery that neuronal dysfunction or degeneration can be caused by mutations in single cellular proteins has given new opportunities to model the underlying disease processes by genetic modification of cells in vitro or by generation of transgenic animals carrying the disease-causing gene. Recent developments in recombinant viral-vector technology have opened up an interesting alternative possibility, based on direct gene transfer to selected subregions or subsets of neurons in the brain. Using the highly efficient adeno-associated virus or lentivirus vectors, recent reports have shown that overexpression of mutated human huntingtin or α-synuclein in neurons in the striatum or substantia nigra induces progressive neuropathology and neurodegeneration, similar to that seen in Huntington's and Parkinson's diseases. Targeted overexpression of disease-causing genes by recombinant viral vectors provides a new and highly flexible approach for in vivo modeling of neurodegenerative diseases, not only in mice and rats but also in primates. (Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Trends in Neurosciences
volume
26
issue
7
pages
386 - 392
publisher
Elsevier
external identifiers
  • wos:000184258600011
  • pmid:12850435
  • scopus:0038015531
ISSN
1878-108X
DOI
10.1016/S0166-2236(03)00164-4
language
English
LU publication?
yes
id
786c8093-9079-4431-b87e-3e6fdf56fe01 (old id 116674)
alternative location
http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=12850435&dopt=Abstract
date added to LUP
2007-07-16 12:49:41
date last changed
2018-01-07 09:10:09
@article{786c8093-9079-4431-b87e-3e6fdf56fe01,
  abstract     = {Defective handling of proteins is a central feature of major neurodegenerative diseases. The discovery that neuronal dysfunction or degeneration can be caused by mutations in single cellular proteins has given new opportunities to model the underlying disease processes by genetic modification of cells in vitro or by generation of transgenic animals carrying the disease-causing gene. Recent developments in recombinant viral-vector technology have opened up an interesting alternative possibility, based on direct gene transfer to selected subregions or subsets of neurons in the brain. Using the highly efficient adeno-associated virus or lentivirus vectors, recent reports have shown that overexpression of mutated human huntingtin or α-synuclein in neurons in the striatum or substantia nigra induces progressive neuropathology and neurodegeneration, similar to that seen in Huntington's and Parkinson's diseases. Targeted overexpression of disease-causing genes by recombinant viral vectors provides a new and highly flexible approach for in vivo modeling of neurodegenerative diseases, not only in mice and rats but also in primates.},
  author       = {Kirik, Deniz and Björklund, Anders},
  issn         = {1878-108X},
  language     = {eng},
  number       = {7},
  pages        = {386--392},
  publisher    = {Elsevier},
  series       = {Trends in Neurosciences},
  title        = {Modeling CNS neurodegeneration by overexpression of disease-causing proteins using viral vectors.},
  url          = {http://dx.doi.org/10.1016/S0166-2236(03)00164-4},
  volume       = {26},
  year         = {2003},
}