mTOR inhibitors may benefit kidney transplant recipients with mitochondrial diseases

Mitochondrial diseases represent a significant clinical challenge. Substantial efforts have been devoted to identifying therapeutic strategies for mitochondrial disorders, but effective interventions have remained elusive. Recently, we reported attenuation of disease in a mouse model of the human mitochondrial disease Leigh syndrome through pharmacological inhibition of the mechanistic target of rapamycin (mTOR). The human mitochondrial disorder MELAS/MIDD (Mitochondrial Encephalopathy with Lactic Acidosis and Stroke-like Episodes/Maternally Inherited Diabetes and Deafness) shares many phenotypic characteristics with Leigh syndrome.

Source: Kidney International - November 21, 2018 Category: Urology & Nephrology Authors: Simon C. Johnson, Frank Martinez, Alessandro Bitto, Brenda Gonzalez, Cagdas Tazaerslan, Camille Cohen, Laure Delaval, Jos é Timsit, Bertrand Knebelmann, Fabiola Terzi, Tarika Mahal, Yizhou Zhu, Philip G. Morgan, Margaret M. Sedensky, Matt Kaeberlein, Chr Tags: Clinical Trial Source Type: research