Non-perturbative stable gene editing enabled by nanostraws

Training cells to fight disease is the future of medicine.

Using gene editing technology, breakthroughs have been made that allow researchers to cure previously uncurable diseases.

Unfortunately, progression has been slow within cell types that are rare and sensitive where current means to bring in the gene editing molecules result in massive cell death, perturbed cell function, and – in the case of stem cells – risk of mutagenesis.

A new methodology to deliver molecules to clinically relevant cells is acutely needed.This project will uniquely enable a nanotechnological approach based on nanostraws for intracellular molecular delivery which is completely non-toxic even to very sensitive primary cells.

The outcomes of this project will provide a pathway to genetically correct and engineer patient specific cells for autologous cell therapies.

New biology will be accessed by enabling sequential delivery of biomolecules without loss of efficiency or cell viability.

The approach will also enable extraction of cytosolic contents allowing for a lysis-free way to establish the success of the gene editing.

The extraction will enable direct determination of gene editing in the cells meant for transplantation, instead of in a sister culture which is the standard today.

In summary, this project will lay the foundation towards a future clinical concept where these nanostructures will be implemented to genetically engineer cells in order to treat diseases.