Please use this identifier to cite or link to this item: http://hdl.handle.net/2440/7747
Citations
Scopus Web of ScienceĀ® Altmetric
?
?
Full metadata record
DC FieldValueLanguage
dc.contributor.authorFerrara, M.en
dc.contributor.authorOcchiodoro, T.en
dc.contributor.authorFuller, M.en
dc.contributor.authorHawthorne, W.en
dc.contributor.authorTeutsch, S.en
dc.contributor.authorTucker, V.en
dc.contributor.authorHopwood, J.en
dc.contributor.authorStewart, G.en
dc.contributor.authorAnson, D.en
dc.date.issued1997en
dc.identifier.citationNeuromuscular Disorders, 1997; 7(5):361-366en
dc.identifier.issn0960-8966en
dc.identifier.issn1873-2364en
dc.identifier.urihttp://hdl.handle.net/2440/7747-
dc.description.abstractSevere progressive fatal neurological degeneration occurs in fucosidosis, a storage disease. Bone marrow transplantation into affected dogs has shown that haematopoietic stem cells can provide enzyme producing daughter cells to the central nervous system, altering disease course. This makes canine fucosidosis an ideal large animal model for gene therapy. Fucosidosis affected allogeneic or autologous canine marrow was transduced ex vivo by cocultivation, then transplanted into fucosidosis affected dogs conditioned with total lymphoid irradiation. The vectors were Moloney murine leukaemia virus based. Transduction efficiency was increased with multiple cytokines in short term marrow culture. Despite high levels of transduction, proviral sequence was detected 2 months post transplant in only one dog. Early or total graft failure occurred in all transplants. We believe lack of engraftment could be caused by differentiation or change of repopulating ability of marrow cells occurring with multiple cytokine mixes in culture media.en
dc.language.isoenen
dc.publisherPERGAMON-ELSEVIER SCIENCE LTDen
dc.subjectHematopoietic Stem Cells; Animals; Dogs; Retroviridae; Fucosidosis; Treatment Failure; Gene Transfer Techniques; Genetic Vectors; Female; Maleen
dc.titleCanine fucosidosis: a model for retroviral gene transfer into haematopoietic stem cellsen
dc.typeJournal articleen
dc.identifier.rmid0030005426en
dc.identifier.doi10.1016/S0960-8966(97)00060-6en
dc.identifier.pubid69432-
pubs.library.collectionPaediatrics publicationsen
pubs.verification-statusVerifieden
pubs.publication-statusPublisheden
dc.identifier.orcidFuller, M. [0000-0001-9092-8942]en
Appears in Collections:Paediatrics publications

Files in This Item:
There are no files associated with this item.


Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.