A study by a team of scientists at the Children’s Research Institute was reported this week in the journal Nature Communications. The study is welcomed by the medical profession as it tests gene therapies in a fully developed human liver. Gene therapy is considered revolutionary as it is designed to treat severe hereditary (genetic) diseases that involve repairing or replacing a faulty gene.
This touches on an issue that researchers face in their effort to bring gene therapies to the clinic. In this era of personalized medicine, there has been a shortage of preclinical liver models that can be used to develop gene editing technologies.
Laboratory pre-testing must take place before a therapy may be used to treat a patient. Note also that lab models should accurately mirror bodily functions and organization of tissue. This is necessary in order to predict results after the patient has been administered therapy.
Then and Now
In the past and due to their complexity, livers were not suitable transplant candidates. They were either discarded or remained on ice and used for research.
With credit to the combined efforts of researchers at the Royal Prince Albert Hospital and CMRI’s Vectorology Research Unit, it is now possible to preserve liver outside the human body and use it for biomedical research.
The CMRI researchers have successfully demonstrated the use of this system to test AAV-based therapeutics before the initiation of clinical studies.
Being able to use the whole human liver is considered revolutionary in gene therapy. Researchers are now able to test the effect of the new therapeutics on the liver. This was not possible in the last decade.
About the Delivery System
The adeno-associated virus (AAV) is considered to be a most efficient delivery system as it is based on a harmless virus that can transport genetic information to human cells.
Leszek Lisowski M.D., senior author of the study, stressed the importance of the new finding, saying that the current generation of viral vectors used for delivery are incapable of meeting the demands of the majority of clinical applications. He further stated that this work will lead to fewer animals being used for research in the future.
The scientists have a goal of developing more effective treatments to fulfill the needs of people with life-threatening inherited disorders.
