The world-renowned reputation of Children’s Medical Research Institute’s Gene Therapy Research Unit has been recognised with the team asked to contribute to a scientific book.

Professor Ian Alexander, Dr Samantha Ginn and PhD student Sharntie Christina were invited to contribute to the book “Curing Genetic Diseases though Gene Reprogramming” from the series Progress in Molecular Biology and Translational Science. The aim of this book is to cover the diversity of applications of "gene reprogramming" for human diseases, including genome editing technology.

Gene therapy involves using genes to treat or cure disease. It may be used to either repair or replace a fault in your DNA.

Dr Ginn said they were asked to write about genome editing in the liver in a chapter summarising the current state of the field. Professor Alexander’s lab specialises in targeting the liver to treat metabolic disorders and has achieved significant success in this area.

“Our group has had a long history in this area but now we’ve set the bar quite high in terms of a therapeutic model. If we can achieve what we are setting out to do, it will be very exciting,’’ Dr Ginn said. “Success will open up a lot of opportunities for the field.’’

Up until now the only way that many children living with liver disorders could survive was to have a liver transplant. However, there is a lack of donors and children still face the possibility of liver rejection and life-long immune suppression. That is why CMRI’s gene therapy team are focused on gene therapy for the liver.

In the book the authors write about their optimism for the future:

“Gene therapy lies at the forefront of modern medicine and, with more than 30 years of incremental development, the field has reached an inflexion point where many difficult or impossible to treat diseases are increasingly falling within therapeutic reach.’’

Dr Ginn said that pioneering trials for haemophilia A and B are already showing clinical success and that their group are working on developing liver-based trials for more challenging diseases.

“Now we are looking at how to move on from gene replacement to genome editing. We want to make permanent changes to the genome so that the therapeutic benefits are maintained for the life of the patient.

“Being asked to take part in this publication is based on the reputation of Ian’s group and everything we’ve been working hard to achieve.’’