Genome editing is the process by which precise genetic changes can be made at exact locations in the genome in living cells. In primary human cells, the preferred method of delivering the donor template DNA, that carry the genetic changes to be introduced, is using viral vectors based on adeno-associated virus (AAV). However, a major drawback of AAV vectors is their inherent restricted carrying capacity of 4.7 kilobases (kb), which limits the applications of genome editing for large or multi-gene genomic integration. This project aims to develop a novel genome editing platform based on multiple AAV donors that allow site-specific integration of large gene cassettes that exceed the capacity of a single AAV donor. Such a system expands the current genetic toolbox for studying gene function, and it enables therapeutic gene editing for a number of diseases.
Rasmus O. Bak is an Associate Professor and an AIAS-COFUND Fellow. He holds a Master’s degree in Nanoscience and a PhD in Biomedicine, both from Aarhus University. Rasmus has returned to Aarhus University from a three-year postdoctoral research fellowship at Stanford University in California where he acquired research skills in the use of CRISPR/Cas9 gene editing in human cells.
Precise CRISPR-mediated genomic integration of large transgenes in human cells
Area of research:
01 Oct 2017 – 10 Dec 2020