Article

A Genetic Model Therapy Proposes a Critical Role for Liver Dysfunction in Mitochondrial Biology and Disease

2020-05-09

Abstract excerpt

The clinical and largely unpredictable heterogeneity of phenotypes in patients with mitochondrial disorders demonstrates the ongoing challenges in the understanding of this semi-autonomous organelle in biology and disease. Here we present a new animal model that recapitulates key components of Leigh Syndrome, French Canadian Type (LSFC), a mitochondrial disorder that includes diagnostic liver dysfunction. LSFC is...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 763db629-6a03-5d74-b80c-17f0a2ba9ced
DOI: 10.1101/2020.05.08.084681

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

A Genetic Model Therapy Proposes a Critical Role for Liver Dysfunction in Mitochondrial Biology and DiseaseDOI 10.1101/2020.05.08.084681Read

A Primer Genetic Toolkit for Exploring Mitochondrial Biology and Disease Using ZebrafishSemantic proximity Read

Disease models of Leigh syndrome: From yeast to organoids.Semantic proximity Read

A primer genetic toolkit for exploring mitochondrial biology and disease using zebrafishSemantic proximity Read

Zebrafish as a Useful Model System for Human Liver DiseaseSemantic proximity Read

Hepatic gene replacement improves energy metabolism and survival in a mouse model of neonatal mitochondrial disease GRACILE syndrome.Semantic proximity Read

Mitochondrial and nuclear genomic responses to loss of LRPPRC expression.Semantic proximity Read

Hepatic gene replacement restores energy metabolism and doubles the survival in mouse model of GRACILE syndrome, a neonatal mitochondrial diseaseSemantic proximity Read

Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transferSemantic proximity Read

Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transferSemantic proximity Read

Biallelic variants in LARS1 induce steatosis in developing zebrafish liver via enhanced autophagySemantic proximity Read

Zebrafish Models of Human Liver Development and DiseaseSemantic proximity Read

LRPPRC mutations cause early-onset multisystem mitochondrial disease outside of the French-Canadian population.Semantic proximity Read