Treating Mendelian diseases with genetic suppressors
Most Mendelian diseases lack effective treatments.
Suppressor genes, which, when mutated, rescue the detrimental effects of a disease mutation, can encode promising alternative drug targets.
Recent years have seen increased development of drugs targeting suppressors.
Genetic suppressors are common and may exist for many Mendelian disease genes.
Systematic identification and validation of suppressor variants could transform therapeutic strategies for rare genetic diseases.
https://www.cell.com/trends/genetics/fulltext/S0168-9525(26)00146-0
