10 July 2023
How PXB-mice with humanized livers enable better translational outcomes for gene-based therapeutics
In recent years, we have seen an explosion in the number of adeno-associated virus (AAV) vector-based gene therapies progressing to clinical trial stages, and subsequently an increasing number of regulatory successes. The reasons for this are clear. The versatility of AAV-based platforms makes them prime technology for targeting a range of tissues and pathologies, through their ability to silence, replace or modify genes.
