Potential of gene therapy for pediatric neurotransmitter diseases: Lessons from Parkinson's disease
Identifieur interne : 001724 ( Main/Exploration ); précédent : 001723; suivant : 001725Potential of gene therapy for pediatric neurotransmitter diseases: Lessons from Parkinson's disease
Auteurs : Un Jung Kang [États-Unis] ; Ken Nakamura [États-Unis]Source :
- Annals of Neurology [ 0364-5134 ] ; 2003.
Abstract
Gene therapy methods have continued to develop rapidly, and many initial limitations that hampered clinical application have been overcome. Thus serious consideration of clinical application of gene therapy is warranted for selected disorders in which the pathogenesis is well defined. Parkinson's disease has been the most extensively studied target of gene therapy for central nervous system disorders and shares many features with pediatric neurotransmitter diseases. Neurotransmitter replacement therapy using catecholamine‐synthesizing genes and delivery of neurotrophic factors such as glial cell line‐derived neurotrophic factors has been successful in animal models of Parkinson's disease. Application of gene therapy for pediatric neurotransmitter diseases will require delineating the optimal set of genes to correct the consequences of the deficiencies. The optimal anatomical targets and proper timing of the gene replacement must be understood. Safety of gene therapy vehicles and the ability to regulate gene expression will be essential for eventual clinical application. Ann Neurol 2003;54 (suppl 6):S103–S109
Url:
DOI: 10.1002/ana.10654
Affiliations:
Links toward previous steps (curation, corpus...)
Le document en format XML
<record><TEI wicri:istexFullTextTei="biblStruct"><teiHeader><fileDesc><titleStmt><title xml:lang="en">Potential of gene therapy for pediatric neurotransmitter diseases: Lessons from Parkinson's disease</title><author><name sortKey="Kang, Un Jung" sort="Kang, Un Jung" uniqKey="Kang U" first="Un Jung" last="Kang">Un Jung Kang</name></author><author><name sortKey="Nakamura, Ken" sort="Nakamura, Ken" uniqKey="Nakamura K" first="Ken" last="Nakamura">Ken Nakamura</name></author></titleStmt><publicationStmt><idno type="wicri:source">ISTEX</idno><idno type="RBID">ISTEX:DA732B3E35D94D547EBE6BED523AD0602211622C</idno><date when="2003" year="2003">2003</date><idno type="doi">10.1002/ana.10654</idno><idno type="url">https://api.istex.fr/document/DA732B3E35D94D547EBE6BED523AD0602211622C/fulltext/pdf</idno><idno type="wicri:Area/Main/Corpus">001193</idno><idno type="wicri:Area/Main/Curation">000F94</idno><idno type="wicri:Area/Main/Exploration">001724</idno></publicationStmt><sourceDesc><biblStruct><analytic><title level="a" type="main" xml:lang="en">Potential of gene therapy for pediatric neurotransmitter diseases: Lessons from Parkinson's disease</title><author><name sortKey="Kang, Un Jung" sort="Kang, Un Jung" uniqKey="Kang U" first="Un Jung" last="Kang">Un Jung Kang</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><placeName><region type="state">Illinois</region></placeName><wicri:cityArea>Departments of Neurology and Neurobiology, Pharmacology, and Physiology, University of Chicago, Chicago</wicri:cityArea></affiliation></author><author><name sortKey="Nakamura, Ken" sort="Nakamura, Ken" uniqKey="Nakamura K" first="Ken" last="Nakamura">Ken Nakamura</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Department of Neurology, University of California, San Francisco, San Francisco</wicri:cityArea></affiliation></author></analytic><monogr></monogr><series><title level="j">Annals of Neurology</title><title level="j" type="sub">Official Journal of the American Neurological Association and the Child Neurology Society</title><title level="j" type="abbrev">Ann Neurol.</title><idno type="ISSN">0364-5134</idno><idno type="eISSN">1531-8249</idno><imprint><publisher>Wiley Subscription Services, Inc., A Wiley Company</publisher><pubPlace>Hoboken</pubPlace><date type="published" when="2003">2003</date><biblScope unit="volume">54</biblScope><biblScope unit="issue">S6</biblScope><biblScope unit="supplement">6</biblScope><biblScope unit="page" from="S103">S103</biblScope><biblScope unit="page" to="S109">S109</biblScope></imprint><idno type="ISSN">0364-5134</idno></series><idno type="istex">DA732B3E35D94D547EBE6BED523AD0602211622C</idno><idno type="DOI">10.1002/ana.10654</idno><idno type="ArticleID">ANA10654</idno></biblStruct></sourceDesc><seriesStmt><idno type="ISSN">0364-5134</idno></seriesStmt></fileDesc><profileDesc><textClass></textClass><langUsage><language ident="en">en</language></langUsage></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Gene therapy methods have continued to develop rapidly, and many initial limitations that hampered clinical application have been overcome. Thus serious consideration of clinical application of gene therapy is warranted for selected disorders in which the pathogenesis is well defined. Parkinson's disease has been the most extensively studied target of gene therapy for central nervous system disorders and shares many features with pediatric neurotransmitter diseases. Neurotransmitter replacement therapy using catecholamine‐synthesizing genes and delivery of neurotrophic factors such as glial cell line‐derived neurotrophic factors has been successful in animal models of Parkinson's disease. Application of gene therapy for pediatric neurotransmitter diseases will require delineating the optimal set of genes to correct the consequences of the deficiencies. The optimal anatomical targets and proper timing of the gene replacement must be understood. Safety of gene therapy vehicles and the ability to regulate gene expression will be essential for eventual clinical application. Ann Neurol 2003;54 (suppl 6):S103–S109</div></front></TEI><affiliations><list><country><li>États-Unis</li></country><region><li>Californie</li><li>Illinois</li></region></list><tree><country name="États-Unis"><region name="Illinois"><name sortKey="Kang, Un Jung" sort="Kang, Un Jung" uniqKey="Kang U" first="Un Jung" last="Kang">Un Jung Kang</name></region><name sortKey="Nakamura, Ken" sort="Nakamura, Ken" uniqKey="Nakamura K" first="Ken" last="Nakamura">Ken Nakamura</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
EXPLOR_STEP=$WICRI_ROOT/Wicri/Sante/explor/ParkinsonV1/Data/Main/Exploration
HfdSelect -h $EXPLOR_STEP/biblio.hfd -nk 001724 | SxmlIndent | more
Ou
HfdSelect -h $EXPLOR_AREA/Data/Main/Exploration/biblio.hfd -nk 001724 | SxmlIndent | more
Pour mettre un lien sur cette page dans le réseau Wicri
{{Explor lien
|wiki= Wicri/Sante
|area= ParkinsonV1
|flux= Main
|étape= Exploration
|type= RBID
|clé= ISTEX:DA732B3E35D94D547EBE6BED523AD0602211622C
|texte= Potential of gene therapy for pediatric neurotransmitter diseases: Lessons from Parkinson's disease
}}
| This area was generated with Dilib version V0.6.23. |  |