Selectively expressed RNA molecules against neuronal diseases

About

Cell targeting defines one of the most challenging and important needs in modern medical treatment approaches in order to affect specifically diseased cells while minimizing side effects for healthy cells. We developed selectively expressed RNA (seRNA) molecules that transfer cell targeting for the very first time from the cell surface of diseased cells to their whole intracellular transcriptome. By interaction with any cell type specific RNA out of this transcriptome, seRNAs change their conformation to switch from a formerly inactive molecular pro-drug to its active form, enabling translation. At the same time, lack of activation in healthy cells prevents seRNA induced protein effector formation. Our research aims to further characterize underlying seRNA functional mechanisms and to tune regulatory aspects. At the same time, we are developing seRNA molecules for reseach as well as preclinical and clinical applications against diseases with highest medical need, such as brain tumors (glioblastoma) or Neuronal Ceroid Lipofuscinosis (NCL).

Research Topics

RNA therapies are one of the most promising approaches in modern medicine. However, their universal functionality in every cell of the human body is both a blessing and a curse, since treatment of diseased cells with RNA drugs come along with massive cytotoxic side effects also in healthy cells.

Contact

Dr. Bernd Hoffmann

IBI-2

Building 02.4W / Room 337

+49 2461/61-6734

E-Mail
Selectively expressed RNA molecules against neuronal diseases
seRNA treatment of glioblastoma in mice results in massive tumor reduction

Selected Publications

Hoffmann, M., Schuele, S., Hoffmann, C., Rastfeld, F., Gerlach, S., Hersch, N., Walter, H.L., Wiedermann, D., Fink, G.R., Merkel, R., Bohlen, H., Rueger, M.A. and B. Hoffmann (2022) Selectively expressed RNA molecules: a new dimension in functionalized cell targeting. bioRxiv. https://doi.org/10.1101/2022.05.10.491309

Last Modified: 22.02.2023