ABOVE: Adeno-associated viruses (AAVs) are commonly used to deliver gene therapies, but they can trigger immune responses that undermine the treatment.
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Gene therapy has the potential to be the ultimate treatment for inherited diseases. Instead of treating the symptoms, it addresses the root cause by replacing a defective gene, and thus its missing or misfit product. Although several promising gene therapies are in late-stage clinical trials, major roadblocks remain. Chief among them is the immune system, which can sabotage the therapies by attacking the viruses that carry them.
Gene therapies such as Spark Therapeutics’s recently approved Luxturna (voretigene neparvovec-rzyl), which cures an inherited form of blindness, rely on engineered viruses to ferry disease-fighting genes to the cells where they’re needed. Adeno-associated viruses (AAVs) are used frequently because they are small and adept at getting into hard-to-reach organs such as the brain, and because they are not harmful to ...
