Background: Antisense oligonucleotides (AON) represent a promising treatment for Duchenne muscular dystrophy (DMD) carrying out-of-frame deletions, but also show limitations. In a completed clinical trial golodirsen, approved by FDA to induce skipping of DMD gene exon 53 in eligible patients, we demonstrated increase in DMD expression and protein production, albeit with inter-patient variability.
Methods: Here, we investigate further the golodirsen mechanism of action using myotubes derived from MyoD transfected fibroblasts isolated from DMD patients at the baseline of the clinical trial SRP-4053.
Eur Phys J C Part Fields
October 2024
The CYGNO experiment aims to build a large ( m ) directional detector for rare event searches, such as nuclear recoils (NRs) induced by dark matter (DM), such as weakly interactive massive particles (WIMPs). The detector concept comprises a time projection chamber (TPC), filled with a He:CF 60/40 scintillating gas mixture at room temperature and atmospheric pressure, equipped with an amplification stage made of a stack of three gas electron multipliers (GEMs) which are coupled to an optical readout. The latter consists in scientific CMOS (sCMOS) cameras and photomultipliers tubes (PMTs).
View Article and Find Full Text PDFCorrection for 'Bio-inspired copper complexes with CuS cores: (solvent) effects on oxygen reduction reactions' by Jordan Mangue , , 2024, , 15576-15582, https://doi.org/10.1039/D4DT01629G.
View Article and Find Full Text PDFThe need for effective alternative energy sources and "green" industrial processes is a more crucial societal topic than ever. In this context, mastering oxygen reduction reactions (ORRs) is a key step to develop fuel cells or to propose alternatives to energy-intensive setups such as the anthraquinone process for hydrogen peroxide production. Achieving this goal using bio-inspired metal complexes based on abundant and non-toxic elements could provide an environmentally friendly option.
View Article and Find Full Text PDFCurr Treat Options Cardiovasc Med
December 2023
Purpose Of The Review: Even as immune checkpoint inhibitors (ICIs) have transformed the lifespan of many patients, they may also trigger acceleration of long-term cardiovascular disease. Our review aims to examine the current landscape of research on ICI-mediated atherosclerosis and address key questions regarding its pathogenesis and impact on patient management.
Recent Findings: Preclinical mouse models suggest that T cell dysregulation and proatherogenic cytokine production are key contributors to plaque development after checkpoint inhibition.