Our lab is currently investigating the use of gene therapy for genetic diseases that affect children, including cystic fibrosis, alpha-1 antitrypsin (AAT) deficiency, type I diabetes, and disorders of fatty acid oxidation. We are studying adeno-associated virus (AAV) vectors and the mechanisms of AAV persistence, since these represent the basis for a more profound understanding of the potential for long-term safe and effective gene therapy. We are currently conducting Phase I trials with rAAV expressing alpha-1antitrypsin in AAT-deficient patients.
Our lab is always interested in possible partnerships with both indivituals and organizations. Some possible partnerships include knowledge exchange, consulting, collaborations with academia and/or industry, sponsored research, spinout activities and reagent exchange. Read more