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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 Baruteau
Published 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.
Keyphrases
  • gene therapy
  • oxidative stress
  • stem cells
  • bone marrow
  • fatty acid