Profil
I am a research scientist with a strong background in developing strategies and applying novel techniques for genome engineering in human cells. I have >17 years of research experience during which I have worked with gene therapy in genetic diseases and immuno-oncology. I have experience with a wide panel of gene expression vectors, such as plasmids, transposons, lentiviral vectors, and adeno-associated viral vectors. In addition, I have expertise in several CRISPR/Cas-based platforms such as knockout, knockin, gene replacement, transcriptional regulation, base editing, and prime editing. I have developed chemically modified guide RNAs for use in the CRISPR/Cas9 system and shown that they can facilitate very high levels of genome editing in primary human hematopoietic stem and progenitor cells (Nature Biotechnology, 2015). I used this platform to develop a potent CRISPR/Cas9-based genome editing strategy for Sickle Cell Disease (Nature, 2016; Nature Protocols, 2018). I have also demonstrated that the system supports integration of large transgenes by sequential homologous recombination (Cell Reports, 2017) and multiplexed genome editing (eLife, 2017). Finally, I have developed precision tools for manipulating transcriptional of individual genes, aka. CRISPRa and CRISPRi, (Genome Research, 2022; Molecular Therapy, 2024), and developed and applied prime editing for liver-directed gene therapy (Molecular Therapy, 2022).

My primary area of responsibility is leading and managing a multidisciplinary research group consisting of students, PhD candidates, postdoctoral researchers, and research assistants. Our research focuses on biomedical applications of CRISPR/Cas-based genetic engineering, with the aim of advancing genome-editing technologies for therapeutic and translational purposes. I am responsible for setting the strategic direction of our research, securing funding, and fostering collaborations both nationally and internationally.

In addition to research leadership, I contribute actively to teaching and supervision in genetics, particularly for medical students. I also coordinate and teach an annual AU Summer University course in genetic engineering, where I provide an in-depth exploration of genome editing techniques, applications, and ethical considerations.

Furthermore, I am engaged in outreach and dissemination activities to communicate our research findings effectively. This includes scientific presentations at conferences, publications in peer-reviewed journals, and interactions with the broader scientific community. I also prioritize public engagement, aiming to make complex genetic research accessible to a wider audience through public lectures, media contributions, and collaborations with policymakers and industry stakeholders.