Genetic medicines could treat certain diseases, however, the current delivery systems available—viral vectors and lipid nanoparticles (LNPs)—pose unique challenges for gene therapy applications. For ...
Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in gene therapy using ... pH found inside cells, demonstrating sufficient ...
Our independent appraisal committee has approved the use of gene editing therapy exagamglogene autotemcel (exa-cel) for use in the NHS in England, providing a potential cure for some people with ...