Mahley · Arteriosclerosis, thrombosis, and vascular biology 2016 · narrative review · n=?

Central Nervous System Lipoproteins: ApoE and Regulation of Cholesterol Metabolism.

Cited 530 times in the scientific literature.

Level 5 - mechanism / opinion, no new human data

Narrative review summarizing molecular and pre-clinical mechanism-based research without primary human data.

PubMed 27174096 · doi:10.1161/ATVBAHA.116.307023 · record verified 2026-08-30

What was done

This narrative review describes the role of apolipoprotein E (apoE) isoforms in central nervous system (CNS) lipid transport and cholesterol homeostasis, and discusses molecular mechanisms and interventions targeting apoE4 structural defects.

What was found

No quantitative clinical numbers are reported in the abstract. ApoE on high-density lipoproteins facilitates CNS cholesterol and phospholipid redistribution for membrane repair. ApoE4 shows lower expression in brain and cerebrospinal fluid, reduced cholesterol and phospholipid secretion, decreased lipid-binding capacity, and increased intracellular degradation in cultured astrocytes and neurons compared to apoE2 and apoE3. ApoE4 dysfunction is driven by conformational instability and an ionic domain interaction between arginine-61 and glutamic acid-255. Disrupting this interaction by gene targeting (arginine-61 to threonine) or small-molecule structure correctors increases apoE4 levels, improves lipid binding, and reduces degradation and neurotoxic fragment formation.

Why it matters

The review outlines the mechanistic basis for apoE4-associated CNS lipid dysregulation and identifies small-molecule structure correctors as potential therapeutic candidates for Alzheimer disease.

Limits

The abstract describes pre-clinical and cellular mechanistic findings without human trial data, sample sizes, or quantitative outcome measures.

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