Reveal the correlations between gene therapy vectors and toxicity

Description

Gene therapy brings great hope to cure many inherited or rare diseases. However, as more and more gene therapies are expected to be approved for clinical trials, new problems may arise. Adeno-associated virus, AAV, does not cause any known disease and are commonly used as vector in gene therapy. But it was found that sometimes it can cause toxicity and inflammation in targeted tissues in animals and humans. A joint-research by City University of Hong Kong (CityU) and Harvard Medical School has revealed that the AAV toxicity is correlated with the cis-regulatory sequences which control gene expression in the virus genome.