Article
SMRT compounds abrogate cellular phenotypes of ataxia telangiectasia in neural derivatives of patient-specific hiPSCs.
Nature communications - 1 Jan 2013
Lee Peiyee, Martin Nathan T, Nakamura Kotoka, Azghadi Soheila, Amiri Mandana, Ben-David Uri, Perlman Susan, Gatti Richard A, Hu Hailiang, Lowry William E
Abstract excerpt
Ataxia telangiectasia is a devastating neurodegenerative disease caused primarily by loss of function mutations in ATM, a hierarchical DNA repair gene and tumour suppressor. So far, murine models of ataxia telangiectasia have failed to accurately recapitulate many aspects of the disease, most not...
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