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.
Precise CRISPR-mediated genomic integration of large transgenes in human cells
Area of research:
1 Oct 2017 – 10 Dec 2020
AIAS-COFUND Marie Skłodowska-Curie fellow
This fellowship has received funding from the European Union’s Seventh Framework Programme for research, technological development and demonstration under the Marie Skłodowska-Curie grant agreement No 609033 and The Aarhus University Research Foundation.