Targeting miR-155 in Peripheral Monocytes for the Treatment of ALS

Posted August 26, 2016
Dr. Howard Weiner, Brigham and Women’s Hospital, Boston, MA

Dr. Howard Weiner

Researchers continue to investigate the dysregulated mechanisms behind ALS pathology.  One growing area of research is the impact of microRNAs in ALS.  MicroRNAs are small non-coding RNA molecules (containing about 22 nucleotides) which can carry out RNA silencing and post-transcriptional regulation of gene expression, leading to various disease processes.  Dr. Howard Weiner and co-investigator Dr. Oleg Butovsky at the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital are using an ALSRP Therapeutic Development Award to investigate one miRNA of interest, miR-155, in the SOD1 mouse model of ALS as well as human sporadic and familial ALS.  In previous research, Dr. Weiner and colleagues discovered that microglia and peripheral monocytes in SOD1 mice demonstrated upregulated miR-155, as did monocytes in the blood from ALS patients.  Given miR-155’s known role in inflammation, Dr. Weiner and colleagues hypothesized that its inhibition, and subsequent suppression of downstream target genes, would rescue ALS pathology.

Successful targeting of miR-155 in the ALS mouse model led these researchers to establish a collaboration with miRagen Therapeutics in 2013 to develop a strategy to target miR-155 as a treatment for ALS.  This collaboration was recently recognized with a Translational Research Advancing Therapy for ALS (TREAT ALS™) grant from the ALS Association to miRagen to advance the development of MRG-107, a synthetic microRNA antagonist of microRNA-155, which was shown to be effective in pre-clinical models of amyotrophic lateral sclerosis (ALS).  Additional findings from Dr. Weiner’s ALSRP award are being prepared for publication.


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