Ex vivo precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases.
Dany P PerocheauSonam GurungLoukia TouramanidouClaire DuffGarima SharmaNeil SebirePatrick F FinnAlex CavedonSummar SiddiquiLisa RicePaolo G V MartiniAndrea FrassettoJulien BaruteauPublished in: F1000Research (2024)
Therapeutic response to gene therapy such as messenger RNA replacement delivered via lipid nanoparticles can be monitored, demonstrating that precision-cut liver sections can be used as a preclinical screening tool to assess therapeutic response and toxicity in monogenic liver diseases.