The UMass Gene Therapy Center has been established around the promise that lies within the application of the recombinant adeno-associated virus (rAAV). These viruses are a diverse class of non-pathogenic, naturally replicated-defective and persistent parvoviruses of humans and related primates, sharing no homology with adenovirus, but rather named as it is because it was initially discovered as a contaminant of adenovirus cultures. The Gene Therapy Center builds up its basic research programs around AAV evolution and biology, novel vector discovery, host –AAV vector interactions. Collaborating with RNA Therapeutics Institute and Stem Cell Centers, the Gene Therapy Center develops its translational research programs on the rAAV-based gene and RNAi therapeutics for treating Cystic Fibrosis, Alpha-Antitrypsin Deficiency, Lysosomal Storage Diseases, Canavan Disease, Retinal and Macular Degeneration and other genetic diseases by either replacing the missing genes or modulating the harmful gene expression.
Importantly, the center is poised to provide a research environment and infrastructure to facilitate scientific interactions between multi-disciplinary basic, biomedical and clinical researchers. This interaction merges the sustained excellence of the UMMS basic science faculty led by Noble Laureate Dr. Craig Mello and other world renowned scientists with the success of the four clinical research centers of excellence on the UMMS campus in the area of Cancer, Diabetes, Heart and Vascular and Musculoskeletal diseases. Ultimately the center serves as a critical translational bridge between bench discoveries in basic biomedical research and healthcare delivery.
Through its Vector Core, the Center produces high quality research grade AAV vectors for both internal and external clients and is in the early phase of creating a human application GMP for use in gene therapy, stem cell, and RNAi clinical trials.
To provide expertise and infrastructure in viral vector development, vector biology, production and applications, to serve research communities at UMMS, State, national and international levels by working collaboratively with basic research and clinical departments and the RNA Therapeutics Institute and Stem cell centers to develop and translate scientific breakthroughs into novel therapeutics for clinical evaluation in patients.