|
rdf:type |
|
|
lifeskim:mentions |
|
|
pubmed:issue |
3
|
|
pubmed:dateCreated |
2000-10-10
|
|
pubmed:abstractText |
Canavan disease (CD) is an autosomal recessive leukodystrophy characterized by deficiency of aspartoacylase (ASPA) and increased levels of N-acetylaspartic acid (NAA) in brain and body fluids, severe mental retardation and early death. Gene therapy has been attempted in a number of children with CD. The lack of an animal model has been a limiting factor in developing vectors for the treatment of CD. This paper reports the successful creation of a knock-out mouse for Canavan disease that can be used for gene transfer.
|
|
pubmed:grant |
|
|
pubmed:language |
eng
|
|
pubmed:journal |
|
|
pubmed:citationSubset |
IM
|
|
pubmed:chemical |
|
|
pubmed:status |
MEDLINE
|
|
pubmed:issn |
1099-498X
|
|
pubmed:author |
pubmed-author:CampbellG AGA,
pubmed-author:CeciJ DJD,
pubmed-author:EzellE LEL,
pubmed-author:MatalonKK,
pubmed-author:MatalonRR,
pubmed-author:NehlsMM,
pubmed-author:PlattK AKA,
pubmed-author:QuastM JMJ,
pubmed-author:RadyP LPL,
pubmed-author:SkinnerH BHB,
pubmed-author:SurendranSS,
pubmed-author:SzucsSS,
pubmed-author:TyringS KSK,
pubmed-author:WeiJJ
|
|
pubmed:issnType |
Print
|
|
pubmed:volume |
2
|
|
pubmed:owner |
NLM
|
|
pubmed:authorsComplete |
Y
|
|
pubmed:pagination |
165-75
|
|
pubmed:dateRevised |
2007-11-14
|
|
pubmed:meshHeading |
pubmed-meshheading:10894262-Amidohydrolases,
pubmed-meshheading:10894262-Animals,
pubmed-meshheading:10894262-Brain,
pubmed-meshheading:10894262-Canavan Disease,
pubmed-meshheading:10894262-Central Nervous System,
pubmed-meshheading:10894262-Cloning, Molecular,
pubmed-meshheading:10894262-Disease Models, Animal,
pubmed-meshheading:10894262-Gene Therapy,
pubmed-meshheading:10894262-Gene Transfer Techniques,
pubmed-meshheading:10894262-Magnetic Resonance Imaging,
pubmed-meshheading:10894262-Mice,
pubmed-meshheading:10894262-Mice, Knockout,
pubmed-meshheading:10894262-Phenotype
|
|
pubmed:articleTitle |
Knock-out mouse for Canavan disease: a model for gene transfer to the central nervous system.
|
|
pubmed:affiliation |
Department of Pediatrics, Children's Hospital, UTMB Galveston, TX 77555-0359, USA. rmatalon@utmb.edu
|
|
pubmed:publicationType |
Journal Article,
Research Support, U.S. Gov't, P.H.S.,
Research Support, Non-U.S. Gov't
|
