Publications

Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR gene. Patients carrying the most common mutation, p.F508del, benefit from the triple therapy Kaftrio®. We genome-edited the commercially available iPSC line PCIi033-A (wild-type CFTR) to generate the subclone P...

Extracellular vesicles (EVs) are key mediators of maternal-foetal communication, regulating placental function and foetal development through the transfer of bioactive molecules. Although placental EVs play a crucial role in placental function during pregnancy, their contribution to foetal develo...

The successful translation of a stem cell-derived product from the laboratory into an approved medicine requires specific scientific, technical, and regulatory understanding. Since this knowledge base is fragmented across websites and publications, the ISSCR has developed a new resource, The Best...

The DMD gene, which encodes the protein dystrophin, is involved in a group of diseases known as dystrophinopathies, which includes Duchenne Muscular Dystrophy (DMD). DMD is a progressive and lethal muscular disorder mainly affecting boys that results from the loss of function of the longer dystro...

West Nile virus (WNV) is a mosquito-borne virus that causes severe neurological disease in humans. Despite substantial advances, our knowledge of the mechanisms involved in damaging the human brain is still limited. To address this gap, we developed a physiologically relevant in vitro model using...