Alzheimer disease (AD) is the most common dementia, with an estimated prevalence of 30 million people worldwide. Currently, there is no effective treatment that delays the onset or slows the progression of AD. β-Amyloid (Aβ), the key component of plaques, is thought to play a pivotal role in the initiation of AD pathogenesis.1 Previously, all clinical trials of Aβ-targeted therapeutics have failed. There are several possible reasons for this, one of which is that the preclinical drug discovery and development have relied mostly on cell lines or transgenic animals that overexpress Aβ. Testing drugs in human neurons was difficult because brain tissue is inaccessible through biopsies. After the finding that somatic cells can be reprogrammed into induced pluripotent stem cells (iPSCs), which can be further modified into specific types of cells,2,3 neurons induced from patients with familial and sporadic AD have been regenerated, characterized,4 and used to study aspects relevant to AD biology such as toxicity of Aβ oligomers.5
Liao F, Holtzman DM. Human Neurons Derived From Induced Pluripotent Stem Cells as a New Platform for Preclinical Drug Screening and Development. JAMA Neurol. 2014;71(12):1475–1476. doi:10.1001/jamaneurol.2014.2802
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