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Review

Nature Reviews Genetics 5, 136–144 (1 February 2004) | doi:10.1038/nrg1268

Using human neural stem cells to model neurological disease

Rebekah J. Jakel , Bernard L. Schneider & Clive N. Svendsen

Although many common neurological diseases can be modelled in rodents, in many cases these animal models do not faithfully reproduce the human syndrome at either the molecular or anatomical levels — perhaps owing to important species differences. The study of diseased human brain tissue is therefore crucial for understanding how mutant proteins or toxins might lead to neuronal dysfunction. Unfortunately, this tissue is both scarce and difficult to manipulate. Human stem cells represent a renewable source of tissue that can generate both neurons and glia. Studies that use human stem cells from diseased tissues or stem cells that have been engineered to express specific mutant proteins promise to provide new insights into the mechanisms that underlie neurological diseases.