Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
4
pubmed:dateCreated
2003-8-6
pubmed:abstractText
Successful gene therapy for lysosomal storage diseases that involve the bone marrow and hematopoiesis is hindered by the hostile microenvironments created by these diseases and the irreversibility of the pathologic manifestations. Of the 40 lysosomal storage diseases, Gaucher disease type I, Niemann-Pick B, and mucopolysaccharidosis type I have hematopoietic involvement, are treatable by enzyme therapy, and respond to bone marrow transplantation. These storage diseases provide the basis for continued development of gene therapy.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Jul
pubmed:issn
1540-3408
pubmed:author
pubmed:issnType
Print
pubmed:volume
2
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
356-62
pubmed:dateRevised
2006-7-31
pubmed:meshHeading
pubmed:year
2003
pubmed:articleTitle
Perspectives on gene therapy for lysosomal storage diseases that affect hematopoiesis.
pubmed:affiliation
Division and Program in Human Genetics, Children's Hospital Medical Center, TCHRF 1042, 3333 Burnet Avenue, Cincinnati, OH 45229, USA. greg.grabowski@cchmc.org
pubmed:publicationType
Journal Article, Review