Mapping and rewiring the MYBPC3 promoter for rescue of haploinsufficiency driven hypertrophic cardiomyopathy
This work presents a variant-agnostic approach to rescue haploinsufficiency by mapping and rationally redesigning the MYBPC3 promoter and identifies synergistic sequence edits that drive robust increases in MYBPC3 expression.
Aaron Renberg, S. Coppersmith, Owen Merritt et al.
· bioRxiv · 0 citations