Gene Therapy Making a Comeback?

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.

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