Dissociation of disease phenotype and allele silencing in hypertrophic cardiomyopathy
Alexandra Dainis, Kathia Zaleta-Rivera, Alexandre J. S. Ribeiro, Andrew Chia Hao Chang, Ching Shang, Feng Lan, Paul W. Burridge, Joseph C. Wu, Alex Chia Yu Chang, Beth L. Pruitt, Matthew Thomas Wheeler, Euan Angus Ashley
This study compares two allele-specific silencing strategies in a human model of hypertrophic cardiomyopathy, finding that reducing both alleles improves contractility more than targeting only the disease allele.
bioRxiv · Jun 12, 2019 · PreprintGenetics