AAV1-CMV-Luc-PEST

The ability to elicit robust and long-term transgene expression in vivo, combined with minimal immunogenicity and virtually no toxicity, are just a few of the features that make recombinant adeno-associated virus (rAAV) vectors well suited for many gene therapy applications. Successful preclinical studies have encouraged the use of rAAV to transfer therapeutic genes to patients in the clinical setting.

The growing interest in AAV-based therapies is due to a range of features that make rAAV vectors well suited for gene transfer approaches: their ability to elicit robust and long-term transgene expression in animals and humans, and their safety profile has been demonstrated in several phase 1/2 trials with no toxicity, no adverse events after administration, and manageable immune responses. They induce efficient and long-term transduction of non-dividing cells, an important aspect of liver gene therapy strategies. Therapeutic genes carried by AAV vectors have been effectively delivered to skeletal muscle, heart, brain, joints, eyes, and liver. The first AAV clinical trial was conducted in patients with cystic fibrosis in the mid-1990s, and today, more than 70 clinical trials have been approved worldwide for a variety of diseases.