Gene therapy has become a powerful instrument for the treatment of many diseases to prevent or stop the disease from progressing.

Dr. George Aslanidi, Associate Professor and head of the Molecular Bioengineering and Cancer Vaccine lab at The Hormel Institute, University of Minnesota, received an award of $670,000 by the Minnesota Partnership for Biotechnology and Medical Genomics along with collaborator Dr. Kah-Whye Peng, Professor of Oncology at Mayo Clinic. They will work together to provide support for clinical applications of gene therapy in the effort to ensure novel cutting-edge technologies are available for patients.

“Gene therapy aims to alter what nature has planned in an effort to change the outcome to a more positive one for patients,” said Dr. Aslanidi, who joined The Hormel Institute in 2017.  “Manipulation of genetic materials such as gene substitution, gene overexpression, or gene ‘editing’ can stop or alter what was destined to be negative for a patient and turn that around for a better life quality.”

Adeno-associated virus (AAV) is a non-pathogenic human parvovirus that has been most widely explored as a virus-based delivery platform for safe and effective gene therapy of numerous inherited or acquired diseases affecting different organs/tissues. The recent success of AAV-based gene therapy for rare eye disease and muscular atrophy sparks interest for use vectors for wide-verity if genetic and other diseases when positive clinical outcome requires genetic manipulation with cell and tissues.

One of the major limitations in AAV technology applications is the bottleneck caused by the current production process and pre-clinical evaluation of the vector purity and activity.  The current collaborative proposal between Dr. Aslanidi  (The Hormel Institute, University of Minnesota) and Dr. Peng (Mayo Clinic) will provide access of researchers from both institutions to the reliable source of AAV for pre-clinical application produced under Good Laboratory Practice (GLP) principles to ensure safety, consistency, and quality of vectors. The ultimate goal of this study will be to provide patients with expanded, innovative cures.

The three-year grant begins in April 2020. The Minnesota Partnership for Biotechnology and Medical Genomics is funded by the Minnesota State Legislature and funds joint projects between University of Minnesota and Mayo Clinic in an effort to fund novel applications of advances in biotechnology, genomics,  proteomics, imaging, bioinformatics and more in an effort to prevent, diagnose or treat disease to support and protect human health.