Human Pluripotent Stem Cell-Derived Neural Cellsas a Relevant Platform for Drug Screening inAlzheimer’s Disease

Abstract

Extracellular amyloid-beta deposition and intraneuronal Tau-laden neurofibrillary tanglesare prime features of Alzheimer’s disease (AD). The pathology of AD is very complex and still notfully understood, since different neural cell types are involved in the disease. Although neuronalfunction is clearly deteriorated in AD patients, recently, an increasing number of evidences havepointed towards glial cell dysfunction as one of the main causative phenomena implicated in ADpathogenesis. The complex disease pathology together with the lack of reliable disease models haveprecluded the development of effective therapies able to counteract disease progression. The discoveryand implementation of human pluripotent stem cell technology represents an important opportunityin this field, as this system allows the generation of patient-derived cells to be used for diseasemodeling and therapeutic target identification and as a platform to be employed in drug discoveryprograms. In this review, we discuss the current studies using human pluripotent stem cells focusedon AD, providing convincing evidences that this system is an excellent opportunity to advance inthe comprehension of AD pathology, which will be translated to the development of the still missingeffective therapies.