Adeno-Associated Virus (AAV), a Promising Gene Therapy Vector.
Gene therapy is a novel therapeutic platform to treat and potentially cure currently intractable disorders. Among the most promising gene delivery vehicles are vectors based on adeno-associated virus (AAV).
AAV is particularly attractive because:
- It is not known to be associated with any disease.
- Recombinant AAV vectors can trigger long-term gene expression even in the absence of genome integration (at least in postmitotic tissues).
- AAV vectors display only limited immunogenicity.
Due to these favorable properties, enthusiasm for gene therapy vectors based on AAV has steadily increased among researchers over the last few years. In fact, as of February 2016, 167 clinical trials using AAV have either been completed or are in progress. Moreover, the treatment of lipoprotein lipase deficiency with an AAV vector has been approved for clinical use in Europe, which is the first approval of gene therapeutic treatment in the Western World.
The Main Focus of Our Research Is to:
- Gain a better understanding of the cellular roadblocks that prevent the efficient transduction of certain cell types by AAV.
- Develop AAV variants that A) efficiently transduce specific tissues and cell types—in particular, cell types of the cardiovascular system and B) show increased resistance to pre-existing neutralizing antibodies against the naturally occurring AAV serotypes.