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  • Patient Specific Induced Pluripotent Stem Cell Models for Spinal Muscular Atrophy (SMA) Jacksonville, Fla.

    The purpose of this study is to perform RNA sequencing on induced pluripotent stem cells (iPSC).  By comparing the gene expression of motor neurons derived from iPSC cells of phenotypically discordant Spinal Muscular Atrophy (SMA) sibling pairs and individual cases of SMA, we hope to identify potential protective gene modifiers of SMA.   Identifying protective gene modifiers will increase our understanding of the molecular pathogenesis of SMA.

Closed for Enrollment