Time to review the year 2009 for cutting edge clinical research. For the field of gene transfer, it has been an annus mirabilis: a year that has seen very encouraging results in a wide variety of human clinical studies, as well as preclinical studies. Indeed, I regret that this blog has only been able to cover a few of the former, and very little of the latter. Here are a few highlights from clinical studies:
• in March 2009, Italian researchers reported major clinical improvement in eight of ten children participating in a gene transfer study involving ADA-SCID. [discussed here]
• in June 2009, researchers at Penn / Scheie Eye Institute reported very encouraging outcomes in three children with hereditary blindness, including evidence of visual recovery. [discussed here]
• in September 2009, researchers reported “marginal effectiveness” in preventing HIV infection for a gene transfer-based vaccine. These findings from this trial (the “RV144 trial”) were unexpected after abysmal trial results involving a related strategy (the STEP trials). These are the first encouraging results from any HIV vaccine study conducted to date. [described here and here].
• in November 2009, researchers at Paris-Necker reported very encouraging outcomes in two children with adreno leukodystrophy who received a vector derived from lentiviruses [discussed here]
The decade began with a series of very inauspicious clinical outcomes in gene transfer, and a sharp abatement in the volume of clinical testing. The decade ends with several highly encouraging results from well designed and executed clinical trials. (photo credit: Xavier Luque 2009)
@Manual{stream2009-75,
title = {Annus Mirabilis for Gene Transfer},
journal = {STREAM research},
author = {Jonathan Kimmelman},
address = {Montreal, Canada},
date = 2009,
month = dec,
day = 29,
url = {https://www.translationalethics.com/2009/12/29/annus-mirabilis-for-gene-transfer/}
}
MLA
Jonathan Kimmelman. "Annus Mirabilis for Gene Transfer" Web blog post. STREAM research. 29 Dec 2009. Web. 11 Feb 2025. <https://www.translationalethics.com/2009/12/29/annus-mirabilis-for-gene-transfer/>
APA
Jonathan Kimmelman. (2009, Dec 29). Annus Mirabilis for Gene Transfer [Web log post]. Retrieved from https://www.translationalethics.com/2009/12/29/annus-mirabilis-for-gene-transfer/
Time to review the year 2009 for cutting edge clinical research. For the field of gene transfer, it has been an annus mirabilis: a year that has seen very encouraging results in a wide variety of human clinical studies, as well as preclinical studies. Indeed, I regret that this blog has only been able to cover a few of the former, and very little of the latter. Here are a few highlights from clinical studies: