[HTML][HTML] Mitochondrial diseases: hope for the future

OM Russell, GS Gorman, RN Lightowlers, DM Turnbull - Cell, 2020 - cell.com
Mitochondrial diseases are clinically heterogeneous disorders caused by a wide spectrum
of mutations in genes encoded by either the nuclear or the mitochondrial genome …

The special considerations of gene therapy for mitochondrial diseases

J Slone, T Huang - NPJ genomic medicine, 2020 - nature.com
The recent success of gene therapy across multiple clinical trials has inspired a great deal of
hope regarding the treatment of previously intractable genetic diseases. This optimism has …

Therapies for mitochondrial diseases and current clinical trials

AW El-Hattab, AM Zarante, M Almannai… - Molecular genetics and …, 2017 - Elsevier
Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders
that result from dysfunction of the mitochondrial oxidative phosphorylation due to molecular …

Pathogenic mitochondria DNA mutations: current detection tools and interventions

MF Mustafa, S Fakurazi, MA Abdullah, S Maniam - Genes, 2020 - mdpi.com
Mitochondria are best known for their role in energy production, and they are the only
mammalian organelles that contain their own genomes. The mitochondrial genome mutation …

Mutations causing mitochondrial disease: What is new and what challenges remain?

RN Lightowlers, RW Taylor, DM Turnbull - Science, 2015 - science.org
Mitochondrial diseases are among the most common and most complex of all inherited
genetic diseases. The involvement of both the mitochondrial and nuclear genome presents …

Clinical trials in mitochondrial disorders, an update

M Almannai, AW El-Hattab, M Ali… - Molecular genetics and …, 2020 - Elsevier
Mitochondrial disorders comprise a molecular and clinically diverse group of diseases that
are associated with mitochondrial dysfunction leading to multi-organ disease. With recent …

Therapeutic prospects for mitochondrial disease

EA Schon, S DiMauro, M Hirano… - Trends in molecular …, 2010 - cell.com
Until even only a few years ago, the idea that effective therapies for human mitochondrial
disorders resulting from the dysfunction of the respiratory chain/oxidative phosphorylation …

[HTML][HTML] Mitochondrial DNA disease—molecular insights and potential routes to a cure

O Russell, D Turnbull - Experimental cell research, 2014 - Elsevier
Mitochondrial DNA diseases are common neurological conditions caused by mutations in
the mitochondrial genome or nuclear genes responsible for its maintenance. Current …

Moving towards clinical trials for mitochondrial diseases

RDS Pitceathly, N Keshavan… - Journal of inherited …, 2021 - Wiley Online Library
Primary mitochondrial diseases represent some of the most common and severe inherited
metabolic disorders, affecting~ 1 in 4,300 live births. The clinical and molecular diversity …

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 …