NEWS
Jul, 2025, New York City: First in the world IRF2BPL-NEDAMSS gene therapy
We are proud to annouce that functionalgenomics.pl is a partner of the first in the world IRF2BPL gene therapy clinical trial at Weill Cornell Medicine, NYC. After succesfull first dose of AAV9 based gene augumentation this technology will be further developed by https://functionalgenomics.pl in collaboration with https://alcyonetx.com Lowell, Massachusetts and NextGen Precision Health, University of Missouri.
Jun, 2025, Berlin: First in the world IRF2BPL-NEDAMSS transgenic mouse model was born!
We are happy to annouce that first in the world in vivo mocel of IRF2BPL (NEDAMSS) was sucessfully generated by functionalgenomics.pl. Such transgenic mouse now cen be used for in vivo phenotypic study, drug testing and gene therapy testing including further development of the next versions of IRF2BPL gene therapy and delivery challange.
Jun, 2025, Berlin: BAYER Foundation winner quest scientist Sofia Notopoulou joining the lab for Winter 2025/2026
Meet the brilliant young researchers who are breaking boundaries as part of Bayer Foundation’s 2025 cohort of scientific fellowship awardees. Their cutting-edge projects in Drug Discovery and Medical Sciences:
Sofia Notopoulou: From Institute of Applied Biosciences, Greece to Pawel Lisowski, Berlin Institute for Medical Systems Biology, Germany
Project: Targeting Parkinson’s disease culprits in patient-derived iNSCs
May, 2025, Berlin: FENS/IBRO Exchange Fellowships Programme Winner
Sofia Notopoulou, CRISPaR: Gene editing to target Parkinson’s disease, Centre for Research and Technology Hellas, Greece to Pawel Lisowski, Berlin Institute for Medical Systems Biology, Germany in the season ov 2025/2026.
Jan, 2025, Berlin: BOOST project for gene replacement gene therapy
We are happy to annouce that we have launched MDC BOOST engineering project aimed in gene therapy technology developement for diseases with complex gene architecture like Huntington’s disease and IRF2BPL related disease under umbrella of BlueRock THERAPEUTICS LP part of BAYER AG responsible for cell and gene therapies technology development .
Aug 22, 2024, Berlin-Düsseldorf: We have impicated new neurodevelopmental mechanism in Huntington's disease.
In the Nature Communication paper by Lisowski et al. 2024, we have implicated a new gene in the progression of Huntington’s disease in a brain organoid model. The gene may contribute to brain abnormalities much earlier than previously thought. Moreover, our genome editing strategies, in particular the removal of the CAG repeat region in the Huntington gene, showed great promise in reversing some of observed developmental defects. This suggests a potential gene therapy approach.
More information can be found:
https://www.nature.com/articles/s41467-024-51216-w
https://www.sciencedaily.com/releases/2024/08/240822125933.htm
https://e3.eurekalert.org/news-releases/1055398
https://e3.eurekalert.org/multimedia/1038832
https://huntingtonsdiseasenews.com/news/proteins-loss-brain-may-cause-huntingtons-long-before-onset/