Estrogen receptor alpha in the hypothalamus is required for the effects of chronic tamoxifen treament on gene expression, thermoregulation, bone, and movement in mice.
Juan Manuel Povedano, Paula Martinez ... Maria A Blasco
Telomerase gene therapy represents a novel effective treatment for pulmonary fibrosis associated with short telomeres by improving pulmonary function, decreasing inflammation and accelerating fiber disappearance in fibrotic lungs.
Genetic and biochemical approaches identify a new component of the cellular signaling machinery driving migration of limb muscle precursor cells during mouse embryogenesis and reveal the underlying molecular mechanism.
Targeted therapies induce an aberrant fucosylation of complex tumor secretomes stimulating the expansion of minority drug-resistant clones and promoting therapy resistance.
AAV-Txnip prolongs vision in mouse models of blindness, enhancing lactate catabolism, mitochondrial health, and ATP production in a condition that likely has a limited glucose supply.
Stephen Johnston, Sarah L Parylak ... Matthew Shtrahman
Future work using recombinant AAV as an experimental tool in the dentate gyrus or as a gene therapy for diseases of the central nervous system should be carefully evaluated.
Normalizing Tcf4 expression during early postnatal development improves behavioral outcomes in Pitt-Hopkins syndrome (PTHS) model mice, suggesting that genetic therapies are feasible in individuals with PTHS.
Global brain transduction of the instability-prone Mecp2 transgene by systemic AAV-PHP.eB administration is safe and effective in protecting male and female Mecp2 mutant mice from the Rett syndrome disease phenotype.