Rapamycin treatment inhibits mTOR activity and preserves ATP levels in neurons derived from induced pluripotent stem cells from a maternally inherited Leigh syndrome patient.
Jamie L Courtland, Tyler WA Bradshaw ... Scott H Soderling
Cellular etiology of a human WASH complex mutation in mice reveals its specific roles in neuronal organelle trafficking and a surprising role in motor impairments.
Treatment of human pluripotent stem cell-derived myotubes with a cocktail of small molecules induces their maturation, as shown by gene expression, biochemical and functional assays.
Cilia R Pothast, Romy C Dijkland ... Mirjam HM Heemskerk
Insight into pre-existing SARS-CoV-2-specific T cell responses caused by heterologous T cell immunity directed against dissimilar epitopes, mediated by a public T cell receptor.
Apparent age-related differences in learning are best accounted for by rapid decay of information in working memory, which is associated with levels of prefrontal glutamate.
The combined use of NAD+ with ribitol or ribose potentiates the rescue of α-dystroglycan functional glycosylation in FKRP-mutant patient-specific iPSC-derived myotubes, representing potential novel treatments for FKRP muscular dystrophies.
Simone Bersini, Roberta Schulte ... Martin W Hetzer
Direct reprogramming of smooth muscle cells from HGPS patients revealed that BMP4 is a key contributor of vascular degeneration and might represent a new therapeutic target.
Dongsheng Guo, Katelyn Daman ... Charles P Emerson Jr
iMyoblasts, a novel iPS-derived PAX3 muscle stem cell for gene editing, muscle engraftment, and stem cell therapeutics to treat muscle injury and muscular dystrophies.