The liver is a key production and processing site that is essential for health. Liver dysfunction
can result in both systemic and liver-specific diseases. To combat these diseases, genetic
approaches have been developed that have high liver tropism and are based on gene
addition/editing or gene silencing. The gene addition/editing approach has yielded
encouraging clinical data on the use of viral vectors in patients with haemophilia, as well as
neuromuscular diseases, and has led to trials for liver-related disorders. However, the …