Small-molecule hypoxia therapy in mitochondrial disease.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 40118030.
- Also identified by DOI 10.1016/j.cell.2025.02.019 and PMC identifier 12260919.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
In this issue of Cell, Blume et al. provide compelling rationale for pursuing pharmacologic optimization of a small-molecule "HypoxyStat," which left-shifts the oxyhemoglobin dissociation curve in red blood cells in an attempt to induce an effective and sustained reduction of chronic tissue hyperoxia in primary mitochondrial disease (PMD) and was well-tolerated and effective for both pre-symptomatic and advanced disease treatment to extend survival and improve neurologic outcomes in a mouse model of Leigh syndrome spectrum.
Medical subject headings
- Mitochondrial Diseases
- Hypoxia