by Medizinische Hochschule Hannover Graphical abstract. Credit: Molecular Therapy (2022). DOI: 10.1016/j.ymthe.2022.07.003 Gene therapies aim to cure severe, barely treatable monogentic diseases caused by a defect in a single gene. Medical hopes are correspondingly high. Some gene therapies have already been approved in Europe—for example, for spinal muscular atrophy (SMA), a congenital neuromuscular disease with severe muscle...
