The group of Joost Verhaagen received a grant from the KNAW-research fund (€250.000) for their research into non-invasive adeno-associated vector technology. For this project, the Verhaagen group will establish a collaborative national network, coordinated by the NIN.
The aim of the project is to generate and disseminate non-invasive adeno-associated viral vectors (AAVs), with cell selective gene targeting properties for application in a range of biomedical projects. To achieve this, the Verhaagen group will team-up with research groups at University Medical Centers/Dutch universities, Sanquin, and two other KNAW-institutes to design, create and apply non-invasive AAV vector(s) for their specific applications.
Adeno-associated viral vectors (AAVs) are powerful tools for gene delivery to the brain, spinal cord and other organs. AAVs enable gene overexpression, gene editing, neural circuit modification by chemo and optogenetics, and assessment of neuronal activity. The remarkable progress that has been made in the field of gene therapy for a number of inherited and acquired diseases is due to the efficiency and safety of the AAV-vector platform. Most applications of AAV rely, however, on invasive surgical injection of the vector into the tissue of interest. Injection of AAVs only result in expression of the gene of interest in small groups of cells near the injection needle. Invasive surgical injection also can lead to bleeding, tissue damage and gliosis.
The Verhaagen group has a long-standing record in generating and applying AAV vectors for gene delivery to the brain. Recently, two new AAV-variants became available that allow non-invasive gene delivery to the brain or to peripheral neurons. These non-invasive AAV-variants represent a significant advance over traditional AAV vectors and, from an animal experimental point of view, these new AAV-variants are an important refinement over AAV vectors that have to be delivered by invasive surgical injection.