Inborn Errors of Metabolism with Ataxia: Current and Future Treatment Options

T Bremova-Ertl, J Hofmann, J Stucki, A Vossenkaul… - Cells, 2023 - mdpi.com
A number of hereditary ataxias are caused by inborn errors of metabolism (IEM), most of
which are highly heterogeneous in their clinical presentation. Prompt diagnosis is important …

Mitochondria transfer-based therapies reduce the morbidity and mortality of Leigh syndrome

R Nakai, S Varnum, RL Field, H Shi, R Giwa, W Jia… - Nature …, 2024 - nature.com
Mitochondria transfer is a recently described phenomenon in which donor cells deliver
mitochondria to acceptor cells,–. One possible consequence of mitochondria transfer is …