Mitochondrial disease in adults: recent advances and future promise

YS Ng, LA Bindoff, GS Gorman, T Klopstock… - The Lancet …, 2021 - thelancet.com
Mitochondrial diseases are some of the most common inherited neurometabolic disorders,
and major progress has been made in our understanding, diagnosis, and treatment of these …

Leigh syndrome: one disorder, more than 75 monogenic causes

NJ Lake, AG Compton, S Rahman… - Annals of …, 2016 - Wiley Online Library
Leigh syndrome is the most common pediatric presentation of mitochondrial disease. This
neurodegenerative disorder is genetically heterogeneous, and to date pathogenic mutations …

Circulating markers of NADH-reductive stress correlate with mitochondrial disease severity

R Sharma, B Reinstadler, K Engelstad… - The Journal of …, 2021 - Am Soc Clin Investig
Mitochondrial disorders represent a large collection of rare syndromes that are difficult to
manage both because we do not fully understand biochemical pathogenesis and because …

[HTML][HTML] Patient care standards for primary mitochondrial disease: a consensus statement from the Mitochondrial Medicine Society

S Parikh, A Goldstein, A Karaa, MK Koenig, I Anselm… - Genetics in …, 2017 - Elsevier
The purpose of this statement is to provide consensus-based recommendations for optimal
management and care for patients with primary mitochondrial disease. This statement is …

Mitochondrial medicine in the omics era

J Rahman, S Rahman - The Lancet, 2018 - thelancet.com
Mitochondria are dynamic bioenergetic organelles whose maintenance requires around
1500 proteins from two genomes. Mutations in either the mitochondrial or nuclear genome …

Leigh syndrome: a tale of two genomes

AB Bakare, EJ Lesnefsky, S Iyer - Frontiers in Physiology, 2021 - frontiersin.org
Leigh syndrome is a rare, complex, and incurable early onset (typically infant or early
childhood) mitochondrial disorder with both phenotypic and genetic heterogeneity. The …

Defective metabolic programming impairs early neuronal morphogenesis in neural cultures and an organoid model of Leigh syndrome

G Inak, A Rybak-Wolf, P Lisowski, TM Pentimalli… - Nature …, 2021 - nature.com
Leigh syndrome (LS) is a severe manifestation of mitochondrial disease in children and is
currently incurable. The lack of effective models hampers our understanding of the …

Molecular basis of Leigh syndrome: a current look

M Schubert Baldo, L Vilarinho - Orphanet journal of rare diseases, 2020 - Springer
Leigh Syndrome (OMIM 256000) is a heterogeneous neurologic disorder due to damage in
mitochondrial energy production that usually starts in early childhood. The first description …

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 …

Leigh syndrome: neuropathology and pathogenesis

NJ Lake, MJ Bird, P Isohanni… - … of Neuropathology & …, 2015 - academic.oup.com
Leigh syndrome (LS) is the most common pediatric presentation of a defined mitochondrial
disease. This progressive encephalopathy is characterized pathologically by the …