Customized gene therapy strategies
Within the EIC Pathfinder Challenge on Cardiogenomics, we are developing novel gene reporter technology and gene editing & delivery methodologies to create innovative solutions for gene therapy.
Using high-throughput gene reporters for alternative splicing isoform expression (EXSISERS) and non-coding RNA expression (INSPECT), we can screen human induced pluripotent stem cells (hiPSC) for aberrations and modulators of these fundamental cellular processes.
Subsequently, using exonuclease-enhanced prime editing (Exo-PE) and machine learning-based binder design, we develop customized genetically controlled solutions to correct specific pathological aberrations.
More Details:
Publications:
Truong, DJ.J., Armbrust, N., Geilenkeuser, J. et al. Intron-encoded cistronic transcripts for minimally invasive monitoring of coding and non-coding RNAs. Nat Cell Biol 24, 1666–1676 (2022). https://doi.org/10.1038/s41556-022-00998-6
Truong, DJ.J., Phlairaharn, T., Eßwein, B. et al. Non-invasive and high-throughput interrogation of exon-specific isoform expression. Nat Cell Biol 23, 652–663 (2021). https://doi.org/10.1038/s41556-021-00678-x
Truong, DJ.J., Geilenkeuser, J., Wendel, S.V. et al. Exonuclease-enhanced prime editors. Nat Methods 21, 455–464 (2024). https://doi.org/10.1038/s41592-023-02162-w
Contact
Gil Westmeyer
Helmholtz Munich
E-Mail
