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Gene Delivery and Gene Editing (Focus Group - GDGE)

(198) Liver-targeting oral lipid nanoparticle for CRISPR/Cas9 therapy in MASLD

Introduction: Metabolic dysfunction-associated steatotic liver disease (MASLD) is a prevalent condition characterized by excessive hepatic lipid accumulation, often progressing to fibrosis and cirrhosis. Current treatments have limited efficacy, emphasizing the need for novel therapeutic strategies. This study presents a liver-targeting oral lipid nanoparticle system designed to enhance gastrointestinal stability, liver targeting, and gene transfection efficiency. By enabling non-invasive CRISPR-based DGAT2 inhibition in the liver, this approach offers a promising strategy for MASLD treatment.

Learning Objectives:

  • Analyze the development of liver-targeting oral lipid nanoparticles for MASLD.
  • Explore CRISPR/Cas9-based DGAT2 inhibition for treating MASLD via oral gene delivery.
  • Evaluate the efficacy of GCGA-LNPs in enhancing GI stability, liver targeting, and gene editing.