PAWEL LISOWSKI RESEARCH WEBSITE

Welcome to functionalgenomics.pl - fearless research initiative commited to cutting edge science for lifelong cure of people suffering from rare neurodegenarative and neurodevelopmental diseases.

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You can contribute to our functionalgenomics.pl mission and help IRF2BPL Support Group Europe continue to grow:

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THE MISSION OF FUNCTIONALGENOMICS.PL IS TO PROVIDE ONE-TIME NEXT GENERATION GENE THERAPIES THROUGHOUT:

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Development of safe, precise and efficient post mitotic neuronal cell type specific gene editing tools.

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In Vivo delivery of therapeutic gene editing technology into the patient central nervous system.

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Application of 3D patient derived brain organoids driven by AI machine learning to support treament decisions in the context of:

high throughput phenotypic screening,
gene editing/gene therapy toolbox evaluation,
drug repurposing for personalized medicine.

About FunctionalGenomics.pl

Functionalgenomics.pl initiative with headquarter in Berlin, Germany

operates within estabilished United States scientific and technological network under research collaboration agreements with following partners: Center for Gene Therapy, Nationwide Children’s Hospital, Columbus; Mayo Clinic Jacksonville; University Of California San Diego (UCSD); Salk Institute, La Jolla; Sanford Health Sieux Falls; Nemours Children’s Hospital Wilmington.

The European network for clinical study includes: Helmholtz Zentrum München; University Medical Center Utrecht; Universitätsmedizin Göttingen; Universität Düsseldorf Faculty of Medicine of the Heinrich-Heine-Universität; MSH Medical School Hamburg; Københavns Universitet.

Models: In paralel we develop largest NEDAMSS patients specific induced pluripotent stem cell (iPSCs)….

RUNNING PROJECTS

Diagnostics and decoding of undiagnosed, rare and ultra rare neurodevelopmental diseases

We investigate the molecular mechanisms of diseases with the special focus on neurodevelopmental, neurological and neuropsychiatric disorders starting with clinical WES suplemented with WGS and extensive functional genomics (long and short reads RNAseq, scRNAseq, spatial transcriptomics). To get insight into molecular mechanisms of particular disease and to prioritize risk variants especially for undiagnosed disorders we apply stem cell based disease modeling and differentiation of the stem cell along neuronal lineage into neurons of interest and brain organoids to study neurodevelopmental processes in details under unique patient-specific genetic background.

RECENT SPECIFIC CLINICAL AND PRE-CLINICAL STUDY AIMS INTO DECODING AND TREATMENT OF IRF2BPL-RELATED DISORDERS (NEDAMSS)

Dear Parents and Relatives of children with IRF2BPL (Interferon Regulatory Factor 2 Binding Protein Like) – related disorders (NEDAMSS):

You can participate in our collaborative network of IRF2BPL research by donating the patients’ cells. For basic information about our IRF2BPL.DE research initiative please visit https://irf2bpl.de/. For cells donation methods and procedures please contact Dr. Pawel Lisowski directly.