Why the study?
Current clinical trials exclude patients with liver fibrosis because an intact liver architecture is considered essential for efficient and safe AAV-mediated gene delivery.
Does liver fibrosis impact the efficiency of AAV-mediated gene transfer to mouse hepatocytes?
Does liver fibrosis impact the efficiency of AAV-mediated gene transfer to mouse hepatocytes?
The AAV-KP1 capsid variant retains transduction efficiency in fibrotic livers, offering a potential solution for gene therapy in patients with liver fibrosis who are currently excluded from clinical trials.
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AAV-KP1 may overcome fibrosis-related transduction loss in mice; leaves open translation to human gene therapy trials.
Ferriero et al. (2025) studied this question.
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