Moving gene therapies from the lab-bench to the hospital bed: the adeno-associated virus as a promising gene therapy vector to treat disease

Adam R Ramzy


Gene therapy is a treatment method to replace or repair mutated or deleted genes to correct genetic disorders. With many such monogenic diseases lacking effective therapeutic approaches, gene therapy offers an exciting avenue to resolve the underlying genetic abnormality. Further, a gene therapy could also address complex multifactorial diseases by targeting a critical pathway. Regardless of the therapeutic goal, an important factor to consider is the challenging step of efficiently and precisely delivering genes of interest to cells of interest. These vectors must ideally evade immune response as they infect cells of interest and be able to infect a wide spectrum of target cells. One such vector that has demonstrated much promise is the adeno-associated virus (AAV). The AAV was discovered over 50 years ago and since has been studied for the treatment of many diseases both in the research laboratory and recently in the clinic. Since the first AAV clinical trial started in 1996 for the treatment of cystic fibrosis, hundreds of trials have examined AAV based therapies for the treatment of hemophilia, rheumatoid arthritis, Duchenne’s muscular dystrophy, Leber’s congenital amaurosis, lipoprotein lipase deficiency, and many others. With the first AAV drug approved in the last four years, we are at a point to gain great insight into the feasibility of this type of gene therapy product. In this review, I discuss the basic structure and design of AAV vectors and review recent advances in AAV technology that is enabling these therapies to reach the clinic.

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