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Comment
. 2024 Oct 8;8(19):5200-5202.
doi: 10.1182/bloodadvances.2024013845.

Adeno-associated virus and hepatocytes: frenemies?

Glenn F Pierce  1
Affiliations
Comment

Adeno-associated virus and hepatocytes: frenemies?

Glenn F Pierce. Blood Adv. .
No abstract available

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Conflict of interest statement

Conflict-of-interest disclosure: G.F.P. reports consultantcy fees from BioMarin, Novo Nordisk, Regeneron, Spark, and St. Jude Children’s Hospital; is a member of the scientific advisory boards of Be Biopharma, Frontera, Metagenomi, and hC Bioscience; and serves on the board of Voyager Therapeutics and the World Federation of Hemophilia.

Figures

None
Multifarious variables causing unpredictable responses to AAV gene therapy. Many variables, known and unknown, contribute to the success of AAV gene therapy and define the benefit-risk balance for individuals and for disease populations., The manufacturing process remains poorly defined, with lot-to-lot variability and inconsistent scale-up. Each recipient has a unique phenotype based upon their genetics and epigenetic influences. Collectively, these variables dictate the range of individual response to gene therapy, from durable therapeutically beneficial effects to suboptimal responses and durability. Predictability of individual responses is not yet possible because biomarkers for host hepatocyte condition and host defenses are not well established. Professional illustration by Patrick Lane, ScEYEnce Studios.
See this image and copyright information in PMC

Comment on

References

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