Format

Send to

Choose Destination
Arch Dis Child. 2007 Nov;92(11):1028-31.

Update on clinical gene therapy in childhood.

Author information

1
Molecular Immunology Unit, Institute of Child Health, University College London, London, UK. w.qasim@ich.ucl.ac.uk

Abstract

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.

PMID:
17954483
PMCID:
PMC2083628
DOI:
10.1136/adc.2006.108787
[Indexed for MEDLINE]
Free PMC Article

Supplemental Content

Full text links

Icon for HighWire Icon for PubMed Central
Loading ...
Support Center