Gene Therapy Center

Director:  Dr. Guangping Gao 

The UMass Gene Therapy Center (UM-GTC) has been constructed around the promise that lies with the application of the recombinant adeno-associated virus (rAAV) system.  Adeno-associated viruses (AAVs) are a diverse class of non-pathogenic, naturally replication-defective and persistent parvoviruses of humans and related primates, sharing no homology with adenovirus, but rather named as it is because it was discovered as a contaminant of adenovirus cultures.

Unlike commonly used adenovirus vectors which still retain at least 80% of viral genes, AAV based vectors are totally gutted by design and have no viral genes retained, making them much safer. For many years, work in this field was restricted to the common laboratory variant, AAV serotype 2, but recent work by the Director of the UM-GTC, Dr. Guangping Gao, has resulted in the discovery of over 100 novel serotypes, many of which may be exploited as novel vectors for more efficient, safe and persistent gene transfer with a variety of cell and tissue tropisms6-8.  Dr. Gao continues to pursue the expansion of the armamentarium of rAAV vectors and to work with other UMass investigators to adapt such vectors for human purposes.   
 
Also part of the UM-GTC is the Flotte laboratory.  Dr. Terence Flotte has pursued clinical translation of gene therapy for many years, having first published successful in vivo use of rAAV2 for cystic fibrosis transmembrane conductance regulator (CFTR) gene transfer in 1993, and first used rAAV2 in humans with CF in 19959.  His laboratory continues to be actively NIH funded and focused on clinical translation of new vectors for genetic interventions of CF, AAT lung disease, AAT liver disease, and disorders of fatty acid oxidation. 
 
There is a broad interface between the technology and science of gene therapy and RNAi therapeutics, and the UM-GTC will continue to work closely with the RNAi Therapeutics Institute as both entities move forward in the new Advanced Therapeutics Cluster.  The UM-GTC focuses on

  1. Continued outreach to UMMS-CTSA investigators in order to make these technologies broadly available to T1-translational researchers (with a special emphasis on the thematic areas of emphasis).
  2. The recruitment of additional faculty members with emphasis in the areas of vector immunology, new virus discovery and clinical translation.