MHH research team finds new AAV vectors for gene therapy in the heart muscle
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 weakness and atrophy. With the help of so-called viral vectors, therapeutic genes are transported directly into the cell as a "drug". The best-known representatives of these vectors, colloquially known as gene taxis, are the so-called adeno-associated viruses (AAV). However, as they are naturally accustomed to doing so, they also target other tissues in addition to the actual target. ...
