Autophagy in Alzheimer's Disease Pathogenesis





R.A. Nixon

NYU School of Medicine/Nathan Kline Institute, 140 Old Orangeburg Road, Orangeburg, NY 10962, USA



Cellular protein turnover is mediated largely by the proteasome and lysosomal system. While proteasome activity declines during aging and Alzheimers disease (AD), the lysosomal system is mobilized in vulnerable neurons early in the disease. Macroautophagy, a major pathway to lysosomes for the turnover of organelles and long-lived proteins, involves the sequestration and degradation of these cytoplasmic constituents within a series of vesicular compartments (autophagic vacuoles or AVs). Macroautophagy is induced early in AD and starting before beta-amyloid deposition in the PS1-APP mouse model of AD, which may initially support regeneration/repair and protect neurons from accumulating cytotoxic factors. We find, however, that neuronal autophagy may ultimately fail in AD. Early AVs (autophagosomes) and late AVs progressively accumulate in high numbers in dendrites of affected neurons and become the principal organelles within dystrophic neurites, suggesting that AV transport and the maturation of AVs to lysosomes are impeded. The lysosomal pathology in AD and beta-amyloid mouse models is potentiated by presenilin-1 mutations (PS1) that cause more severe early-onset forms of AD. Fibroblasts from patients with PS1-FAD also accumulate AVs abnormally when macroautophagy is induced. Surprisingly, however, the turnover of proteins by macroautophagy, as measured by metabolic labeling, is markedly reduced in PS-FAD fibroblast lines and nearly completely blocked in blastocysts from PS1/2 gene deleted mice, indicating that PS may play a role in macroautophagy and that FAD-causing mutations of PS confer a loss of macroautophagic function. Slowing autophagic/lysosomal protein degradation in the brains of PS/APP mice by inhibiting cathepsins in vivo accelerates neurodegeneration, possibly by impeding turnover of abnormal and potentially toxic proteins and releasing cathepsins. Finally, purified AVs are highly enriched in presenilin 1, nicastrin, and presenilin dependent gamma-ecretase activity and contain the other components needed for A-beta generation. Inducing or inhibiting macroautophagy in intact cells by modulating mTOR kinase induces parallel changes in the extent of macroautophagy induction, AV proliferation, and A-beta production. Our results, therefore, link amyloidogenic and cell survival pathways through macroautophagy, which is activated and possibly dysfunctional in AD. Supported by the NIA and Alzheimers Association.




Key words: lysosomes, proteasome, cathepsins, Alzheimer's disease, amyloid







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