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Epilepsy and Gene Therapy: Resculpturing Synaptic Transmission with Neuropeptides

Kokaia, Merab LU and Toft Sörensen, Andreas LU (2009) In Encyclopedia of Basic Epilepsy Research p.1430-1434
Abstract
One of the most promising and novel strategies to interfere with neurological disease processes is gene therapy using recombinant adeno-associated viral (rAAV) vectors. Such a rAAV-based gene delivery approach is rapidly advancing towards clinical trials. In this regard, neuropeptide Y (NPY) gene transduction into the brain tissue has attracted particular interest due to its potential to regulate and perhaps even ameliorate epileptic conditions. NPY gene transduction by viral vectors in epileptogenic regions of the brain can effectively suppress seizures in animals. The mechanisms underlying the seizure-suppressant effects of an NPY transgene are not well understood; in particular, under which circumstances transgene NPY is released, and... (More)
One of the most promising and novel strategies to interfere with neurological disease processes is gene therapy using recombinant adeno-associated viral (rAAV) vectors. Such a rAAV-based gene delivery approach is rapidly advancing towards clinical trials. In this regard, neuropeptide Y (NPY) gene transduction into the brain tissue has attracted particular interest due to its potential to regulate and perhaps even ameliorate epileptic conditions. NPY gene transduction by viral vectors in epileptogenic regions of the brain can effectively suppress seizures in animals. The mechanisms underlying the seizure-suppressant effects of an NPY transgene are not well understood; in particular, under which circumstances transgene NPY is released, and whether and how it acts on synaptic transmission within the area of viral vector transduction are not known. These questions are of fundamental importance not only for the implementation of such a gene therapy approach in clinical trials with patients, but also for our general understanding of how transgene neuropeptides may act in the brain. (Less)
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author
organization
publishing date
type
Chapter in Book/Report/Conference proceeding
publication status
published
subject
keywords
Activity-dependent release Gene therapy Hippocampus Long-term potentiation (LTP) Neuropeptide Y Recombinant adeno-associated viral (rAAV) vector Subiculum Synaptic plasticity Synaptic transmission Whole-cell patch-clamp
in
Encyclopedia of Basic Epilepsy Research
editor
Schwartzkroin, Philip A.
pages
1430 - 1434
publisher
Elsevier
DOI
10.1016/B978-012373961-2.00109-0
language
English
LU publication?
yes
id
ce7af1fe-9e72-457c-be00-d35c1f0c8434 (old id 1466489)
date added to LUP
2012-10-30 15:22:11
date last changed
2016-09-23 15:15:31
@inbook{ce7af1fe-9e72-457c-be00-d35c1f0c8434,
  abstract     = {One of the most promising and novel strategies to interfere with neurological disease processes is gene therapy using recombinant adeno-associated viral (rAAV) vectors. Such a rAAV-based gene delivery approach is rapidly advancing towards clinical trials. In this regard, neuropeptide Y (NPY) gene transduction into the brain tissue has attracted particular interest due to its potential to regulate and perhaps even ameliorate epileptic conditions. NPY gene transduction by viral vectors in epileptogenic regions of the brain can effectively suppress seizures in animals. The mechanisms underlying the seizure-suppressant effects of an NPY transgene are not well understood; in particular, under which circumstances transgene NPY is released, and whether and how it acts on synaptic transmission within the area of viral vector transduction are not known. These questions are of fundamental importance not only for the implementation of such a gene therapy approach in clinical trials with patients, but also for our general understanding of how transgene neuropeptides may act in the brain.},
  author       = {Kokaia, Merab and Toft Sörensen, Andreas},
  editor       = {Schwartzkroin, Philip A.},
  keyword      = {Activity-dependent release Gene therapy Hippocampus Long-term potentiation (LTP) Neuropeptide Y Recombinant adeno-associated viral (rAAV) vector Subiculum Synaptic plasticity Synaptic transmission Whole-cell patch-clamp},
  language     = {eng},
  pages        = {1430--1434},
  publisher    = {Elsevier},
  series       = {Encyclopedia of Basic Epilepsy Research},
  title        = {Epilepsy and Gene Therapy: Resculpturing Synaptic Transmission with Neuropeptides},
  url          = {http://dx.doi.org/10.1016/B978-012373961-2.00109-0},
  year         = {2009},
}