[Marchetto MC, Brennand KJ, Boyer LF, Gage FH. Induced pluripotent stem cells (iPSCs) and neurological disease modeling: progress and promises. Hum Mol Genet. 2011 Oct 15;20(R2):R109-15.]
The systematic generation of neurons from patients with neurological disorders can provide important insights into disease pathology, progression and mechanism. This review will discuss recent progress in modeling neurodegenerative and neurodevelopmental diseases using induced pluripotent stem cells (iPSCs) and highlight some of the current challenges in the field. Combined with other technologies previously used to study brain disease, iPSC modeling has the promise to influence modern medicine on several fronts: early diagnosis, drug development and effective treatment.
“In addition, mouse models available to study neurological diseases are limited and usually do not fully recapitulate the human neural phenotype. […] Many mouse models and postmortem tissue studies have provided insight into the pathogenesis of PD; however, the former consistently fail to recapitulate the cardinal features of PD and the latter are end-stage representations. […] Similar to PD, before reprogramming technology, the study of AD was severely limited due to the lack of relevant mouse models. […] Clinical trials based on ALS mouse models have largely failed, suggesting a need for the exploration of new ALS models. […] The above-mentioned hereditability, the suggested increase in prevalence and the current lack of early biological markers, relevant mouse models and effective treatments make ASD an attractive disease for future modeling with iPSC. […]”