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Chunk #0 — INTRODUCTION

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Gain of toxic apolipoprotein E4 effects in human iPSC-derived neurons is ameliorated by a small-molecule structure corrector.
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The complexity and multifactorial etiology of Alzheimer’s disease (AD) pose unique challenges for studying its pathogenesis and developing therapies. Efforts to target AD-related pathways have shown promise in animal studies, only to fail in human trials1,2. There is a pressing need to identify novel mechanisms and therapeutic targets for AD using human model systems, such as human neurons.