Linked Life Data

7584122

Source:http://linkedlifedata.com/resource/pubmed/id/7584122

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
7
pubmed:dateCreated
1995-12-18
pubmed:abstractText
Metachromatic leukodystrophy (MLD) is an inherited metabolic disease which is characterized by deficient activity of arylsulfatase A (ASA). This deficiency causes progressive accumulation of cerebroside sulfate (CS) in oligodendrocytes (OLs) in the brain resulting in dysmyelination. We are developing approaches for treating MLD based on direct delivery of ASA genes to brain. In this paper, we report a recombinant adenovirus (Adex1SRLacZL) that efficiently transduces OLs. Moreover, transduction and expression of the human transgene was efficient in primary fibroblasts from MLD patients exposed to similar recombinant adenovirus carrying the ASA gene (Adex1SRASA) In addition, the expressed ASA protein was correctly targeted to lysosomes and was immunoreactive to anti-ASA antibody. The enzymatic activity of ASA in MLD patient cells is corrected from the deficient state to 2.3 to 5.0 times the activity of normal control cells. These results demonstrate the correction of ASA deficiency by a recombinant adenovirus that is potentially useful for transferring the gene to brain.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Sep
pubmed:issn
0969-7128
pubmed:author
pubmed:issnType
Print
pubmed:volume
2
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
443-9
pubmed:dateRevised
2006-11-15
pubmed:meshHeading
pubmed:year
1995
pubmed:articleTitle
Successful transduction of oligodendrocytes and restoration of arylsulfatase A deficiency in metachromatic leukodystrophy fibroblasts using an adenovirus vector.
pubmed:affiliation
Department of Pediatrics, Jikei University School of Medicine, Tokyo, Japan.
pubmed:publicationType
Journal Article, Research Support, Non-U.S. Gov't