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This article reviews the presentation given at the 2023 annual meeting of the Society of Toxicologic Pathology (STP) on liver toxicity observed with adeno-associated viral vector (AAV) gene therapy.
This review summarises the approved rAAV-vector based gene therapies available that are associated with hepatotoxicity, proposed mechanisms of liver injury, challenges in management with pragmatic recommendations into...
The aim of this review is to describe the risk of liver injury according to the indication for gene therapy and the short- and long-term management currently proposed to prevent or correct liver reactions in clinical ...
While significant progress has been made to manage immune-related liver enzyme elevations following sys-temic AAV delivery in humans, recent clinical trials utilizing high vector doses have highlighted a new challenge...
As mentioned above, the immunogenicity of AAV gene therapy may lead to safety concerns, which warrants further investigations and cautious study design. Yet, AAV can also be used for delivery of biologic therapeutics.
The liver toxicity seen in the trial differs from cases linked to other therapies—and Astellas sees potential for transformative clinical benefits.
Gene therapy can cause liver injury with a very wide spectrum of phenotypic expression and severity, with transaminitis being the most common. The mechanisms of hepatotoxicity associated with gene therapy are heteroge...
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.
Hepatotoxicity is the most common side effect actually observed in the clinic. In at least some cases, this is driven by cytotoxic T lymphocyte responses to transduced liver cells — the main...
This paper describes our current understanding of the biologic underpinnings of how liver health affects hemophilia A gene therapy, and provides practical clinical guidance for assessing, monitoring, and managing live...
