https://medicalxpress.com/news/2024-03- ... liver.html
by Children's Medical Research Institute
In a worldwide first-of-its-kind study published in the journal Nature Communications this week, a team of scientists from Children's Medical Research Institute (CMRI) have tested novel gene therapies in a whole human liver, with the goal of developing more effective treatments for life-threatening inherited diseases.
Gene therapy is a revolutionary approach to treating serious genetic diseases that most commonly involves replacing or repairing a faulty gene. The most efficient delivery systems today are those based on a harmless virus named adeno-associated virus (AAV) that has a natural ability to carry genetic information into human cells.