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The sources discuss induced pluripotent stem cells (iPSCs) and their application in modeling and treating neurological diseases like Alzheimer's disease (AD), Parkinson's disease (PD), amyotrophic lateral sclerosis (ALS), and multiple sclerosis. iPSCs offer a significant advancement over previous methods like animal models, which often fail to fully replicate human disease complexities, and postmortem tissue analysis, which is limited in its ability to study dynamic cellular processes. Derived from adult somatic cells like skin cells, iPSCs can be "reprogrammed" to act like embryonic stem cells, giving them the ability to differentiate into various cell types, including the neural cells implicated in neurological disease.
This technology allows researchers to study disease mechanisms and potential treatments in a human-specific context. For example, by generating iPSC-derived neurons from patients with a particular disease, researchers can observe disease-relevant phenotypes—like protein aggregation in neurons with the TDP-43 M337V mutation associated with ALS—and test the effects of different drugs on these phenotypes. This is illustrated in sources and which provide examples of iPSC models used to study various neurological diseases and the specific phenotypes observed in each.
The sources emphasize that the ability to create patient-specific neurons using iPSCs paves the way for personalized medicine in treating neurological diseases. Researchers can create personalized iPSC-derived neurons to study how individuals with the same disease might respond differently to drugs, ultimately enabling the development of more effective and tailored treatments. This is particularly important given the limitations of current treatment options for neurodegenerative diseases, which are often focused on managing symptoms rather than addressing the underlying causes.
