Article
A screen for regulators of survival of motor neuron protein levels.
Nature chemical biology - 19 Jun 2011
Makhortova Nina R, Hayhurst Monica, Cerqueira Antonio, Sinor-Anderson Amy D, Zhao Wen-Ning, Heiser Patrick W, Arvanites Anthony C, Davidow Lance S, Waldon Zachary O, Steen Judith A, Lam Kelvin, Ngo Hien D, Rubin Lee L
Abstract excerpt
The motor neuron disease spinal muscular atrophy (SMA) results from mutations that lead to low levels of the ubiquitously expressed protein survival of motor neuron (SMN). An ever-increasing collection of data suggests that therapeutics that elevate SMN may be effective in treating SMA. We execut...
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