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Gene Therapy Grows Up

Gene Therapy Grows Up

Gene therapy is almost 30 years old with only a handful of approved products across the world and none in the United States. Thus, it should come as no surprise that the history and development of gene therapy has had many twists and turns. Recently, in Clinical and Translational Science, Keeler, ElMallah, & Flotte expertly review the fascinating history of gene therapy and provide key insight into future directions. The first human gene transfer experiment was in 1989. The subsequent road was long and arduous to approved and marketed projects. It wasn’t until 2013 that the first gene therapy was approved in Europe, Alipogene tiparvovec, GlyberaR®, a recombinant adeno-associated virus (rAAV) product for lipoprotein lipase deficiency. There are currently still no approved gene therapies in the United States, although a rAAV gene therapy for a congenital form of blindness is currently under review.  

One lesson learned is that the success and effectiveness of gene therapy is directly correlated to improvements in nonviral gene transfer methods as well as viral gene transfer vectors. It is all about the delivery. The history of gene therapy has mirrored a variety of technologies to effect gene transfer including by physical and chemical nonviral methods as well as various viral vectors, used both ex vivo and in vivo. Currently, approved therapies all reflect the utility of viral vectors for gene therapy. 

After almost 30 years, the hope for gene therapy remains bright. The tantalizing possibility of treating both genetic and other non-genetic disease with gene therapies is ever closer to a reality. In the future, we can expect the incorporation of new molecular tools – such as RNAi and CRISPR/Cas9 – promise to add new options to gene therapeutics. Read through all the exciting possibilities in Keeler, ElMallah, & Flotte.

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