Article

Lipid-based nanoparticles deliver mRNA to reverse the pathogenesis of lysosomal acid lipase deficiency in a preclinical model

2024-12-08

Abstract excerpt

Lysosomal acid lipase (LAL) is the only known enzyme that degrades cholesteryl esters (CEs) and triglycerides (TGs) in the lysosomes. LAL deficiency (LAL-D) results in hepatosplenomegaly with extensive accumulation of CEs and TGs, and can in the most severe cases be a life-threatening condition in early infancy. Using messenger ribonucleic acid (mRNA) for protein replacement is an innovative approach for the treat...

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Literature Corpus work: 1b3357e4-f4ff-5af5-800e-75812b593f08
DOI: 10.1101/2024.12.07.627318

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