Authors
Creative Biogene
Summary
Gene transfer vectors based on adeno-associated virus (AAV) have been the subject of intense research interest from the gene therapy field since being first described in the early 1980s. The result has been the ongoing development of an increasingly powerful gene transfer system that is presently showing exciting therapeutic promise in animal models and early phase clinical trials. A key step in AAV vector development was the discovery that the prototypic AAV2 vector genome could be cross-packaged into the capsids of multiple different AAV isolates from a diversity of species. This process, known as pseudo-serotyping, has proven to be immensely powerful, conferring novel tropism and biology on individual genome/capsid configurations, and greatly improving the utility and flexibility of the AAV vector system.
Introduction
The ability to generate recombinant AAV particles lacking any viral genes and containing DNA sequences of interest for diverse therapeutic applications has proven to be one of the safest strategies for gene therapies (Figure 1). Especially, an increasing number of phase I–III clinical trials using AAV vectors have yielded promising results. For example, in clinical trials for familial lipoprotein lipase (LPL) deficiency, an AAV1‑based vector encoding the gain‑of‑function LPLS447X variant resulted in persistent gene expression and protein activity, which led to sustained decreases in the incidence of pancreatitis. Other monogenic disorders in which AAV vectors have shown safety and efficacy include Leber’s congenital amaurosis type 2, haemophilia B12 and choroideremia11. In parallel to successes with monogenic disorders, AAV vectors have also been applied to idiopathic diseases.
For over a decade, QVirus™ Platform at Creative Biogene has always optimized our AAV service systems. To fulfill its role as a recombinant viral expression vector, the original wild type AAV has been extensively modified to contain only the essential genes required for viral packaging. The AAV capsid proteins dictate the viral tropism and scientists have adopted this property to construct recombinant AAV for targeting specific types of mammalian cells.
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