Basic Research Workshop: A New Era of Gene Editing for Liver Disease

The future is bright for the continued development of novel gene therapy approaches in liver disease. But, a deeper understanding of off-target effects and immune responses generated in host cells by gene carriers is needed to fully realize their potential. This workshop will provide an overview of current and emerging therapeutic gene editing strategies for liver diseases. Speakers will discuss how novel genome editing techniques and designer nucleases offer the ability to carry out sophisticated gene function studies in vivo. In addition, the workshop will look at how the refinement of delivery systems is paving the way for the translation of gene therapy from bench to bedside.