Genetic suppressors as new therapeutic targets for Mendelian diseases.

Genetic suppressors may provide new therapeutic targets for Mendelian diseases by counteracting disease-causing mutations.

• Why it matters: Most Mendelian diseases currently lack treatments that address their genetic causes, highlighting the need for novel therapeutic strategies.
• What they did: The authors review the prevalence of potential suppressors, describe systematic identification approaches, and discuss their therapeutic relevance, emphasizing advances in genome-wide screening and machine learning.
• The result: Identifying genetic suppressors could expand treatment options and improve outcomes for individuals with inherited disorders, with accelerated discovery expected due to technological advances.