Welcome to the Gruntman Lab for Gene Therapy  

 

Our Lab

Gene therapy treatment for generic diseases that affect children

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.

 

Partner with Us

Partnership opportunities

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  

 

   

We believe that great scientific discoveries happen when people work together.

Let's have the next big breakthrough together!

 

 Explore our Research Partnership Opportunities