In a First, Children with Rare Genetic Diseases Get Mitochondrial Transplants from Their Mothers

December 21, 2022

(STAT News) – Currently, no cure exists. But in the last seven years, a group of doctors and researchers in Israel have been working on a potential treatment that takes advantage of mitochondria’s enduring capacity to slip between cellular life forms. It involves augmenting patients’ hematopoietic stem cells — which give rise to all the different kinds of blood cells — with healthy mitochondria donated by their mothers. On Wednesday, the Israeli team reported in Science Translational Medicine that the approach, tried for the first time in six children, proved safe and showed small but significant signs of improvement in symptoms. (Read More)