Low Density Lipoprotein Receptor mRNA exosomes

Low Density Lipoprotein Receptor (LDLR) mRNA exosomes are a novel gene therapy approach for treating familial hypercholesterolemia (FH), particularly homozygous FH (HoFH). This therapy involves encapsulating LDLR mRNA into exosomes, which are small extracellular vesicles (30-150 nm in diameter) that can deliver bioactive substances to target cells. The exosomes are engineered by forcing expression of LDLR in donor cells, resulting in exosomes containing LDLR mRNA. When administered via tail vein injection, these exosomes primarily target the liver, where the mRNA is translated into functional LDLR protein. This restores receptor expression in LDLR-deficient models, effectively reducing lipid deposition in the liver, lowering serum LDL-cholesterol levels, and reducing atherosclerotic plaques and inflammation.

Other names

LDLR mRNA-loaded exosomesExoLdlr