Mutations in SCN1A, the gene encoding voltage-gated sodium channel NaV1. 1, cause a
spectrum of epilepsy disorders that range from genetic epilepsy with febrile seizures plus to …

Industrial sectors perform toxicological assessments of their potential products to ensure
human safety and to fulfill regulatory requirements. These assessments often involve animal …

Congenital human cytomegalovirus (HCMV) infection causes a broad spectrum of central
and peripheral nervous system disorders, ranging from microcephaly to hearing loss. These …

Over 1250 mutations in SCN1A, the Nav1. 1 voltage-gated sodium channel gene, are
associated with seizure disorders including GEFS+. To evaluate how a specific mutation …

Neural cells differentiated from pluripotent stem cells (PSCs), including both embryonic stem
cells and induced pluripotent stem cells, provide a powerful tool for drug screening, disease …

Fragile X syndrome (FXS) is the most frequent cause of inherited intellectual disability and
autism. It is caused by the absence of the fragile X mental retardation 1 (FMR1) gene …

Introduction Human induced pluripotent stem cells (hiPSCs) offer great promise for
regenerative therapies or in vitro modelling of neurodegenerative disorders like Parkinson's …

Neurodevelopmental disorders (NDDs) arise from the disruption of highly coordinated
mechanisms underlying brain development, which results in impaired sensory, motor and/or …

The use of transplanting functional neural stem cells (NSCs) derived from induced
pluripotent stem cells (iPSCs) has increased for the treatment of brain diseases. As such, it is …

Mucopolysaccharidosis type I (MPS I) is a metabolic disorder characterized by a deficiency
in α-l-iduronidase (IDUA), leading to impaired glycosaminoglycan degradation. Current …