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Full-Text Articles in Medicine and Health Sciences
Effect Of The Butyrate Prodrug Pivaloyloxymethyl Butyrate (An9) On A Mouse Model For Spinal Muscular Atrophy., Jonathan D. Edwards, Matthew E.R. Butchbach
Effect Of The Butyrate Prodrug Pivaloyloxymethyl Butyrate (An9) On A Mouse Model For Spinal Muscular Atrophy., Jonathan D. Edwards, Matthew E.R. Butchbach
Department of Pediatrics Faculty Papers
Spinal muscular atrophy (SMA) is an early-onset motor neuron disease that leads to loss of muscle function. Butyrate (BA)-based compounds markedly improve the survival and motor phenotype of SMA mice. In this study, we examine the protective effects of the BA prodrug pivaloyloxymethyl butyrate (AN9) on the survival of SMNΔ7 SMA mice. Oral administration of AN9 beginning at PND04 almost doubled the average lifespan of SMNΔ7 SMA mice. AN9 treatment also increased the growth rate of SMNΔ7 SMA mice when compared to vehicle-treated SMNΔ7 SMA mice. In conclusion, BA prodrugs like AN9 have ameliorative effects on SMNΔ7 SMA mice.
Proteomic Assessment Of A Cell Model Of Spinal Muscular Atrophy., Chia-Yen Wu, Dosh Whye, Lisa Glazewski, Leila Choe, Douglas Kerr, Kelvin H Lee, Robert W Mason, Wenlan Wang
Proteomic Assessment Of A Cell Model Of Spinal Muscular Atrophy., Chia-Yen Wu, Dosh Whye, Lisa Glazewski, Leila Choe, Douglas Kerr, Kelvin H Lee, Robert W Mason, Wenlan Wang
Department of Pediatrics Faculty Papers
BACKGROUND: Deletion or mutation(s) of the survival motor neuron 1 (SMN1) gene causes spinal muscular atrophy (SMA), a neuromuscular disease characterized by spinal motor neuron death and muscle paralysis. Complete loss of the SMN protein is embryonically lethal, yet reduced levels of this protein result in selective death of motor neurons. Why motor neurons are specifically targeted by SMN deficiency remains to be determined. In this study, embryonic stem (ES) cells derived from a severe SMA mouse model were differentiated into motor neurons in vitro by addition of retinoic acid and sonic hedgehog agonist. Proteomic and western blot analyses were …