I worked for a gene therapy startup a number of years ago. It was a very exciting concept then as now. Gene therapy experienced a series of setbacks that nearly extinguished interest in the field in the late ’90s and early 2000’s.
I have recently come across a couple of examples of success of gene therapy projects. Both are ex-vivo corrections of Mendelian gene disorders and both provide hints that the promise of gene therapy may some day be realized.
The first from Cartier and colleagues (Science 326: 818, 2009) described the correction of X-linked adrenoleukodystrophy in two boys using autologous hematopoietic cells carrying a functional ABCD1 gene introduced with a lentivirus vector. The gene therapy appears to have succeeded out to 2 years.
The second report was recently published in Nature from Cavazzana-Calvo et al. (Nature 467:318, 2010). The report describes correction of beta-thalassemia by autologous transplantation of gene-corrected hematopoietic cells. In this case, an 10-year old patient has been able to forego transfusions for two years post-treatment.
I’m glad that our optimism of 15 years ago was not unfounded. We should look for additional successes from gene therapy in the next few years.