April 1, 2024Journal of Viral HepatitisOpen Access

The complex, confusing and poorly understood immune responses to AAV‐mediated gene transfer in haemophilia—Is more or less immunosuppression required?

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Authors

ETEdward G. D. TuddenhamGoodyear (United Kingdom)GFGraham R. FosterUniversity of North Carolina at Chapel Hill

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Implication

Expert review assesses complex immune barriers to AAV vectors in haemophilia, highlighting the debate over whether to intensify or reduce immunosuppression.

Key Points

AAV-mediated gene transfer for haemophilia A and haemophilia B faces major efficacy limits due to complex host immune responses targeting transduced hepatocytes.
Immune suppression yields only partial efficacy across clinical trials, as innate pathways, adaptive responses, and cytokine-mediated inflammation persist.
Review of therapeutic strategies highlights the debate on whether intensified immunosuppression or altered vector delivery can sustain factor VIII and IX expression.

Cite This Study

Tuddenham et al. (2024) studied this question.

synapsesocial.com/papers/68e71176b6db64358768a6cb https://doi.org/10.1111/jvh.13934

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Implication

Expert review assesses complex immune barriers to AAV vectors in haemophilia, highlighting the debate over whether to intensify or reduce immunosuppression.

The complex, confusing and poorly understood immune responses to AAV‐mediated gene transfer in haemophilia—Is more or less immunosuppression required?

Discussion

Member takes

Implication

Key Points

Cite This Study

The complex, confusing and poorly understood immune responses to AAV‐mediated gene transfer in haemophilia—Is more or less immunosuppression required?

Discussion

Member takes

Implication

Key Points

Cite This Study

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