Reposted by Nechama Wieder
*A new pre-print on increasing gene expression!* 🧬📈
doi.org/10.64898/202...
doi.org
Modulating splicing in five prime 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 ...