Thursday, August 23, 2012
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Induced pluripotent stem
(iPS) cell-derived motor neurons from patients with familial amyotrophic
lateral sclerosis (ALS) for in vitro therapeutic screening
In vitro studies suggest iPS cell-derived motor neurons from
patients with familial ALS could be used to screen for new therapeutics. In
cultured iPS cell-derived motor neurons from patients with familial ALS
carrying TAR DNA
binding protein 43
mutations, levels of TDP-43 protein and cytosolic aggregates were greater
than those in iPS cell-derived motor neurons from healthy controls. In the
ALS patient-derived motor neurons, the histone
acetyltransferase inhibitor anacardic
acid reversed the disease phenotype. Next steps include testing
the safety and efficacy of anacardic acid in additional cellular and animal
Published online Aug. 23, 2012
Patent application filed
covering prophylactic and therapeutic applications of anacardic acid;
provisional application filed covering screening applications; available for
Egawa, N. et al. Sci.
published online Aug. 1,
Contact: Haruhisa Inoue, Japan Science and Technology Agency,
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