Gene therapy for liver disease advances to human trials with the help of adeno-associated viral vectors. The research may aid in the treatment of hemophilia. Pictured, blood cells. Photo by royaltystockphoto/Shutterstock

NEW ROCHELLE, N.Y., Dec. 30 (UPI) -- Liver-directed gene therapy using adeno-associated viral, or AAV, vectors to treat diseases like hemophilia have moved on to human testing.

The research, conducted by Lisa Kattenhorn and co-authors from Dimension Therapeutics in Cambridge, Mass., show the potential for additional technological improvements to expand the therapeutic applications of gene therapy to treat liver disease.


The field of AAV gene therapy has progressed significantly over the past decade with the use of new capsid serotype and organ-specific promoters, scientists have developed an increased understanding of the immune response to AAV in liver-directed therapy.

"AAV-based gene therapy to the liver has been a platform for transformational new therapies for genetic diseases such as hemophilia and inborn errors of metabolism," Dr. Terence R. Flotte, editor-in-chief of Human Gene Therapy, Celia and Isaac Haidak Professor of Medical Education and Dean Provost, and Executive Deputy Chancellor at the University of Massachusetts Medical School, said in a press release. "The review by Dr. Kattenhorn and colleagues provides an excellent overview of the current best knowledge in this area."

A number of clinical trials are planned and ongoing in liver-directed therapy in hemophilia A and B, as well as other liver disorders.

Researchers plan to explore areas of future development including re-administration of AAV gene therapy and minimizing the risk of hepatocellular carcinoma.

The study was published in Human Gene Therapy.