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Lancet Neurol. 2014 Mar;13(3):319-29. doi: 10.1016/S1474-4422(13)70276-X. Epub 2014 Feb 17.

Targeting the β secretase BACE1 for Alzheimer's disease therapy.

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Lerner Research Institute, Cleveland Clinic, Cleveland, OH, USA.
Department of Cell and Molecular Biology, The Feinberg School of Medicine, Northwestern University, Chicago, IL, USA. Electronic address:


The β secretase, widely known as β-site amyloid precursor protein cleaving enzyme 1 (BACE1), initiates the production of the toxic amyloid β (Aβ) that plays a crucial early part in Alzheimer's disease pathogenesis. BACE1 is a prime therapeutic target for lowering cerebral Aβ concentrations in Alzheimer's disease, and clinical development of BACE1 inhibitors is being intensely pursued. Although BACE1 inhibitor drug development has proven challenging, several promising BACE1 inhibitors have recently entered human clinical trials. The safety and efficacy of these drugs are being tested at present in healthy individuals and patients with Alzheimer's disease, and will soon be tested in individuals with presymptomatic Alzheimer's disease. Although hopes are high that BACE1 inhibitors might be efficacious for the prevention or treatment of Alzheimer's disease, concerns have been raised about potential mechanism-based side-effects of these drugs. The potential of therapeutic BACE1 inhibition might prove to be a watershed in the treatment of Alzheimer's disease.

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