GeneticsbioRxivPreprint
Dissociation of disease phenotype and allele silencing in hypertrophic cardiomyopathy
Alexandra Dainis, Kathia Zaleta-Rivera, Alexandre J. S. Ribeiro…·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.
DOI 10.1101/642421