Q J Med 2001; 94: 337-339
© 2001 Association of Physicians
Editorial |
Human gene therapy: are we still expecting too much, too soon?
Department of Medicine, Cambridge University, Addenbrooke's Hospital, Cambridge
With the publication of the first complete draft of the human genome,1 we have for the first time a wild-type sequence for very nearly every human gene. Although we are still in the early stages of mastering the technology required to extract the maximum scientific benefit from this vast databank, it is already clear that it will make a contribution to our understanding of biology of unrivalled breadth and magnitude. We can expect yet a further increase in the pace of discovery in the new ‘post-genomic era’.
Even before the whole genomic sequence became available, human molecular genetics had uncovered the fundamental inborn genetic variation which accounts for disease susceptibility in a wide range of diseases, particularly those for which errors in a single gene are largely responsible. With these advances has come the possibility of identifying individuals who will suffer a particular disease in the earliest stages of pregnancy.
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