PT  - JOURNAL ARTICLE
AU  - Waseem Qasim
AU  - H Bobby Gaspar
AU  - Adrian J Thrasher
TI  - Update on clinical gene therapy in childhood
AID  - 10.1136/adc.2006.108787
DP  - 2007 Nov 01
TA  - Archives of Disease in Childhood
PG  - 1028--1031
VI  - 92
IP  - 11
4099  - http://adc.bmj.com/content/92/11/1028.short
4100  - http://adc.bmj.com/content/92/11/1028.full
SO  - Arch Dis Child2007 Nov 01; 92
AB  - The successful use of gene therapy to correct rare immune system disorders has highlighted the enormous potential of such therapies. We review the current state of gene therapy for childhood immune system disorders, and consider why these conditions have been particularly amenable to genetic correction. As with all emerging therapies, there have been unexpected side effects and their underlying mechanisms are the subject of intense research. Minimising such risks through improved vector design will play an important role in developing the next generation of gene based therapies and extending their applicability.