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The goal of innovation in treating diseases is to provide a long-lasting solution. For rare diseases such as sickle cell disease (SCD) and hemophilia, this can mean reducing the number of complications or even increasing life expectancy. One of the innovations that is having increasing attention is gene therapy. Gene therapy entails substituting flawed genes with normal ones by utilizing vectors derived from the outer shells of viruses, retaining the inherent properties of being able to target and enter specific cells. The modified gene is placed within this shell. Gene therapy can target germline or somatic cells - the latter being the most commonly used. The process for gene therapy is in-vivo or ex-vivo, depending on whether the transduction of the cells happens within or outside the body. Clinical trials in gene therapy have progressed tremendously, and even a few have reached approval by the FDA - but none yet for SCD or hemophilia. For both these diseases, the current treatments provide symptomatic relief but not long-lasting benefits. Currently, there are several gene therapy clinical trials ongoing for both conditions. This paper focuses on published results of sickle cell diseases and hemophilia and examines whether they are pointing towards short-term benefits or whether the effect is long-term.
By: Sanjitha Sadhneni