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Jacob A Hoffman @jahoffman1.bsky.social · 19/11/2024

Ever wondered how gene therapies know where to go? Screening AAV vector libraries across different species I uncovered evidence of altered #glycan profiles between mouse, NHP and human liver, with implications for translation of #AAV #genetherapy pubmed.ncbi.nlm.nih.gov/39001819/ Enjoy!

pubmed.ncbi.nlm.nih.gov

Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity - PubMed

Effective use of adeno-associated viruses (AAVs) for clinical gene therapy is limited by their propensity to accumulate in and transduce the liver. This natural liver tropism is associated with severe...

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