Modulating splicing in 5ʹ untranslated regions to treat rare haploinsufficient disease
Rare genetic disorders collectively impact over 300 million people worldwide, yet around 95% have no specific treatments. For the many rare disorders caused by haploinsufficiency, effective therapies need to upregulate protein expression. However, therapeutic upregulation is often not straightforward. Increasing...
Eloise S. Beer Wells, L. De Conti, Hyung Chul Kim et al.
· Genome Medicine · 0 citations