Differential expression of HEBP2 in amyotrophic lateral sclerosis.

Abstract: Neurodegenerative diseases that affect the motor neurons, including amyotrophic lateral sclerosis (ALS), have little treatment options and are generally rapidly fatal (1, 2). We harnessed the power of unbiased, whole transcriptome differential gene expression analysis, utilizing primary patient cells and tissues to discover genes whose expression defines ALS using published and public data (3, 4). We found significant differential expression of HEBP2, encoding heme-binding protein 2, in the motor neurons of patients with ALS. HEBP2 was also differentially expressed in the induced pluripotent stem cell (iPSC)-derived motor neurons of patients with ALS. HEBP2 transcript was present at significantly lower levels in ALS patient motor neurons as compared to control motor neurons. These analyses will begin to define the transcriptional landscape of ALS.