Nanoparticles as solution to target gene therapies to the heart

Pitch door wetenschappers van het UMC Utrecht.

Gene therapies are a promising treatment option for genetic heart muscle diseases such as PLN. In such a disease, gene therapy can address the cause and then prevent the development of symptoms.

This is unlike current treatments. These are aimed only at reducing symptoms. The underlying disease process continues. A major challenge for this group of therapies is that it is difficult to target them specifically to the heart. In addition, we want to prevent these therapies from entering other organs in high doses.

Preventing damage organs

The high doses in gene therapy can cause severe damage in the liver, for example, and thus lead to liver dysfunction. This, of course, is not what we want. Currently, “benign” viruses are being used to specifically deliver a gene therapy to heart muscle cells.

Addressing the heart

The downside of this is that it activates the immune system in some of the people, which clears the viruses with gene therapy before it can reach the heart. For this reason, several researchers in Utrecht are working on other ways to target gene therapy to the heart.

One such way is nanoparticles: a technology explained in this pitch. This pitch will be made by Professor Joost Sluijter, a leading researcher who has long been involved in PLN research. Next to him is Dr. Nazma Ilahibaks. She talks about an innovative way to target gene therapies specifically to heart muscle cells.

We currently have several gene therapies available for PLN, but we do not yet have a good way to administer them specifically in the heart. As a result, they cannot yet be used in patients. We hope nanoparticles can provide a solution to this problem!

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