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:
https://lup.lub.lu.se/record/116674
- author
- Kirik, Deniz
LU
and Björklund, Anders
LU
- organization
- publishing date
- 2003
- 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
- 2016-04-01 16:17:22
- date last changed
- 2022-01-28 18:39:26
@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}},
doi = {{10.1016/S0166-2236(03)00164-4}},
volume = {{26}},
year = {{2003}},
}