Development and validation of novel gene editing delivery vehicles ‘nanoblades’ retargeted to T cells and hematopoietic stem cells

This project is situated in the field of novel modalities to deliver gene therapy. The objectives are: (1) To pseudotype nanoblades (gene editing delivery vehicles) using novel paramyxovirus glycoproteins (gps) in order to redirect them to T Cells or HSCs. (2) To evaluate the targeting efficiency of these nanoblades in vitro. (3) Up-scaling and improving the production of the novel nanoblades. (4) in vivo evaluation of novel nanoblades in patient cells and humanised mice. The project will involve wet lab work including viral vector production and testing in relevant animal models.

A successful project will result in: (1) Nanoblades efficiently targeting T cells and HSCs. (2) Optimised production protocol for retargeted nanoblades.

Enrolment in Doctoral School: Université Côte d’Azur (Nice)

Planned secondments:

• KU Leuven, Belgium (M19-22): Upscaling and QC of novel nanoblade production; 

• University of Tübingen, Germany (28-29): evaluation of nanoblades in OoC models; 

• Miltenyi Biotech, Germany (EP, M38): Working in GMP environments.