Engineering chromosomes for delivery of therapeutic genes.
The ability to create fully functional human chromosome vectors represents a potentially exciting gene-delivery system for the correction of human genetic disorders with several advantages over viral delivery systems. However, for the full potential of chromosome-based gene-delivery vectors to be realized, several key obstacles must be overcome. Methods must be developed to insert therapeutic genes reliably and efficiently and to enable the stable transfer of the resulting chromosomal vectors to different therapeutic cell types. Research to achieve these outcomes continues to encounter major challenges; however recent developments have reiterated the potential of chromosome-based vectors for therapeutic gene delivery. Here we review the different strategies under development and discuss the advantages and problems associated with each.
Irvine
Danielle V
DV
Chromosome Research Group, Murdoch Childrens Research Institute, Royal Children's Hospital, Department of Paediatrics, University of Melbourne, Flemington Road, Parkville 3052, Australia.
Shaw
Margaret L
ML
Choo
K H Andy
KH
Saffery
Richard
R
D000818:Animals; D002461:Cell Line, Transformed; D002877:Chromosomes, Human / Q000235:genetics*; D018014:Gene Transfer Techniques; D005818:Genetic Engineering / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage*; D006801:Humans; D008845:Microinjections; D014162:Transfection