The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect.
Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within the first
weeks or months of life is inevitable. Some diseases can be managed using conventional medicines whereas others are, as yet, untreatable.
In this review we consider the application of early intervention gene therapy in neonatal and fetal preclinical studies. We appraise
the tools of this technology, including lentivirus, adenovirus and adeno-associated virus (AAV)-based vectors. We highlight the application
of these for a range of diseases including hemophilia, urea cycle disorders such as ornithine transcarbamylase deficiency, organic
acidemias, lysosomal storage diseases including mucopolysaccharidoses, glycogen storage diseases and bile metabolism. We conclude by
assessing the advantages and disadvantages associated with fetal and neonatal liver gene transfer
