miRNA for PAH Treatment

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​Stratified miRNA Target for Early PAH Treatment Pulmonary Arterial Hypertension (PAH) is a serious condition that can lead to heart failure and affects over million people each year. The condition has a poor prognosis and left untreated, those with PAH have only a 35% of a five year survival. Traditional therapies have not significantly improved these survival rates and there is an urgent need for novel therapies. More women get PAH than men but until recently the reason for this has not been clear. miRNAs are a new candidate drug call, which hold great promise. Researchers at the University of Glasgow have validated that the 5HT1B receptor is a target for a miRNA and results have been confirmed in human cell systems. Of special interest is that in a female patient with the R899X mutation, 5HT1B levels were highest and miR96 expression extremely low.  It was observed that elevated oestrogen levels in female mice account for the suppression of miR96 expression as pharmaceutical depletion of oestrogen reverses the phenotype and increases miR96.  The miR96 gene has oestrogen-responsive regions and oestrogen induces decreased miR96 expression in mouse and human cells.   Key Benefits Results have been confirmed that the 5HT1B receptor is a target for a miRNA in human pulmonary artery smooth muscle cell systems (PASMC). A miRNA mimic could be a novel and effective strategy to treat PAH.