The vicissitudes of gene therapy

[摘要] All fields of endeavour have their ups anddowns, but gene therapy arguably suffersmore than most. The predominant early concern was safety. In the broad sweep of eventssince the first approved gene transfer to ahuman in 1989,1 there have been two majorsetbacks as a result of well-publicized patientdeaths. The first, that of Jesse Gelsinger in1999,2 deflated what had until then been adecade of increasing optimism and achievement that saw the approval of approximately485 gene therapy trials. After a hiatus, progress resumed only for momentum to bereversed once again, this time by the occurrence of leukaemia in children receivinggene therapy for severe combined immunodeficiency disease (SCID).3 Nevertheless, atabout this time, the Chinese authoritiesapproved the world’s first gene therapeutic,Gendicine (Shenzhen SiBiono GeneTech,Shenzhen, China), for the treatment of headand neck cancer (Table I).4 Approval of a genetherapy by Western countries did not occuruntil 2012, when the European MedicinesAssociation (EMA) authorized Glybera(UniQure, Amsterdam, The Netherlands) forlipoprotein lipase deficiency.

[学科分类] 骨科学