French and Belgian hepatologists from the Association Française pour l’Etude du Foie (AFEF) (French Association for the Study of the Liver) have taken stock of what is known about the liver toxicity observed with certain gene therapies used in particular for hereditary diseases:
- a fairly exhaustive inventory of gene therapy products and the diseases concerned has been drawn up,
- including experience acquired in humans in the fields of haemophilia and neuromuscular diseases (infantile spinal muscular atrophy, myotubular myopathy, etc.),
- the mechanisms leading to direct or indirect hepatocyte toxicity have been well studied,
- as have the clinical pictures encountered, ranging from a transient rise in transaminases to acute and, in rare cases, fatal liver failure,
- At this stage, corticosteroid therapy remains the best treatment, both preventively and curatively.
This article is accompanied by recommendations that may be useful for clinicians who have to inject a gene therapy product.
