Monday, October 3, 2011
This article addresses the implications of a completed Human Genome Project for gene therapy. It uses the case of cystic fibrosis, the "most common life-threatening single-gene disorder," to illustrate some hurdles to effective gene therapy. The benefits of using a human promoter sequence (rather than that of a virus) in the therapeutic plasmid are discussed, as well as how details down to the plasmid's exact DNA sequence can greatly affect the efficacy of the therapy. Dr. Deborah Gill of the UK Cystic Fibrosis Gene Therapy Consortium says that the HGP has "provided all the tools to make things really easy" and has "revolutionized gene therapy on a support basis." If greater efficiency in gene transfer and a longer response are priorities, how can the information of the HGP be exploited to indeed "revolutionize gene therapy"?