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 >16 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, Sept. 2015). I used this platform to develop a potent CRISPR/Cas9-based genome editing strategy for Sickle Cell Disease (Nature, Nov. 2016; Nature Protocols, Jan. 2018). I have also demonstrated that the system supports integration of large transgenes by sequential homologous recombination (Cell Reports, July 2017) and multiplexed genome editing (eLife, Sept. 2017). Finally, I have developed precision tools for manipulating transcriptional of individual genes, aka. CRISPRa and CRISPRi, (Genome Research, 2022), and developed and applied prime editing for liver-directed gene therapy (Molecular Therapy, 2022)
