Recently, we showed therapeutic effects of the combination of lisinopril (L), an angiotensin converting enzyme (ACE) inhibitor, and spironolactone (S), an aldosterone antagonist, in mice lacking dystrophin and haploinsufficient for utrophin (utrn+/−;mdx, het mice); both cardiac and skeletal muscle function and histology were improved when these mice were treated early with LS. The gene discussed is UTRN; the disease is Leigh syndrome.