Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.
Scientists from the Cardiovascular Research Center at the University of Virginia School of Medicine and Astrazeneca plc have developed a new mouse model of cardiovascular disease associated with genetic variations of cholesterol metabolism. The animal allows in vivo studies of myocardial infarction, plaque rupture and stroke.
Epigenetic silencing could prevent the production of proteins that cause pathologies. CHARM (coupled histone tail for autoinhibition release of methyltransferase), a DNA methylation-based editor, suppressed transcription of prion proteins in the brains of mice.
