Mitochondrial Myopathies in 2025: What Treatments Actually Work
A comprehensive review of pathophysiology, genetics, and emerging therapies for mitochondrial myopathies, spotlighting elamipretide and gene therapy.
268 articles in this topic
A comprehensive review of pathophysiology, genetics, and emerging therapies for mitochondrial myopathies, spotlighting elamipretide and gene therapy.
A phase 2b trial finds allogeneic MSC infusions improved 6-minute walk distance by 63 meters vs placebo at 9 months in frail older adults.
Cytosine base editing achieved 62โ89% efficiency fixing a hyperinflammatory disease gene, but revealed distinct genotoxicity risks across cell types.
Harvard-developed ENTER nanoparticles outperform lipid transfection reagents in delivering mRNA, CRISPR, proteins, and siRNA to diverse cell types.
A special issue overview surveys three decades of peptide-based materials research, spotlighting applications from drug delivery to regenerative medicine.
A landmark clinical trial transplanted iPSC-derived dopamine neurons into Parkinson's patients using only tacrolimus โ with no significant immune rejection.
Researchers 3D-printed silicone scaffolds seeded with human iPSC-derived spinal neural progenitor cells, transplanted them into transected rat spinal cords, and observed significant functional recovery at 12 weeks.
Adding methylene blue to ropivacaine in a serratus anterior plane block significantly prolongs pain relief up to 72 hours post-op.
Researchers erased imprinting marks in mouse sperm using dCas9-TET1, then traced how methylation partially recovered โ fingering H3K9me3 as the key mediator.
New research in Nature Aging suggests tissue mechanical softness is a critical factor enabling cellular regeneration, with major implications for aging biology.
Harvard's CellCartographer uses machine learning to identify optimal transcription factor combinations for rapid, high-efficiency cell reprogramming.
Kyoto researchers created jawbone-like organoids from human iPSCs, recapitulating mandibular development and modeling fragile bone disease.