A research team at the German Heart Centre at Charité (DHZC), led by Prof. Maximilian Emmert, has collaborated with Swiss biotechnology company DiNAQOR to develop a fascinating technical approach that allows gene therapies to be specifically concentrated in a single organ. The results were published in the journal JACC: Basic to Translational Science.
In the study, the method was examined using the kidney as an example. For treatment, the organ is temporarily disconnected from the rest of the blood circulation and supplied via its own circulation. This procedure is known as a closed-loop perfusion system. Gene therapy can thus be administered directly into the isolated organ, while other organs are hardly exposed.
‘We were able to show that a single organ in a living organism can be targeted very precisely and highly effectively – without the risks of systemic distribution,’ says Maximilian Emmert. ‘This opens up new perspectives for gene therapies, but also for many other active substances such as chemotherapies or antibodies, which could be administered in a locally concentrated manner while at the same time being significantly gentler on the rest of the body.’
This approach significantly increases the local concentration of the active substances used, while at the same time reducing the strain on other tissues. The kidney served as an easily accessible model organ in this study. However, the basic principle of isolated organ perfusion can also be applied to other organs.
The procedure is still in the preclinical stage. Further studies are required before it can be used on humans.
Abbreviated version of a press release from the DHZC. Read the full release here.
