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<i>In vitro</i> and <i>ex vivo</i> evaluation of second‐generation histone deacetylase inhibitors for the treatment of spinal muscular atrophy

18 May 2006

Abstract excerpt

Among a panel of histone deacetylase (HDAC) inhibitors investigated, suberoylanilide hydroxamic acid (SAHA) evolved as a potent and non-toxic candidate drug for the treatment of spinal muscular atrophy (SMA), an alpha-motoneurone disorder caused by insufficient survival motor neuron (SMN) protein levels. SAHA increased SMN levels at low micromolar concentrations in several neuroectodermal tissues, including rat...

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PMID: 16805808
DOI: 10.1111/j.1471-4159.2006.03868.x

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