Gene therapy for disorders affecting children progress and potential

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dc.contributor.authorAnson, D.
dc.contributor.authorFletcher, J.
dc.date.issued2007
dc.descriptionCopyright © 2007 The Authors The definitive version is available at www.blackwell-synergy.com
dc.description.abstractFor over two decades gene therapy has been actively pursued as a treatment modality for the inherited diseases that affect the paediatric population, however, it is still to make a real impact in the clinic. There are many reasons for this including inadequate technology and a lack of understanding of the biological complexities that impact on the efficiency of gene delivery and its outcomes, both positive and negative. However, recent progress is now addressing these issues and indicates that these problems can be overcome, and that gene therapy will play a significant role in the treatment of at least some of these disorders. This review will first give a short overview of relevant gene delivery technologies, what strategies can be used and which diseases are potential targets for gene therapy, and then illustrate several specific diseases for which gene therapy is actively being developed.
dc.description.statementofresponsibilityDonald S Anson and Janice M Fletcher
dc.identifier.citationJournal of Paediatrics and Child Health, 2007; 43(5):323-330
dc.identifier.doi10.1111/j.1440-1754.2007.01074.x
dc.identifier.issn1034-4810
dc.identifier.issn1440-1754
dc.identifier.urihttp://hdl.handle.net/2440/45482
dc.language.isoen
dc.publisherBlackwell Publishing Asia
dc.source.urihttp://www.blackwell-synergy.com/doi/pdf/10.1111/j.1440-1754.2007.01074
dc.subjectHumans
dc.subjectGenetic Diseases, Inborn
dc.subjectRisk Factors
dc.subjectPedigree
dc.subjectPediatrics
dc.subjectChild
dc.subjectSouth Australia
dc.subjectGenetic Therapy
dc.titleGene therapy for disorders affecting children progress and potential
dc.typeJournal article
pubs.publication-statusPublished

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