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Delivering therapeutic molecules into cells – advances for regenerative medicines


Centre researchers at the University of Nottingham and Keele University have developed an efficient delivery method to deliver molecules inside of cells as part of the UK’s Regenerative Medicine Platform.  The technology uses small proteins to target heparin sulphate (the cell’s sugar coating) and can deliver peptides, recombinant proteins, nucleic acids, nanoparticles and antibodies within the cell. The GET (GAG-binding enhanced transduction) system, presents a novel approach which will facilitate advances in gene editing, could be exploited for controlling stem cell fate (differentiation) and could also be utilised to load specialised particles to enable in vivo cell tracking.

Published in Proceeding of the National Academy of Sciences (PNAS) this highly controlled intracellular delivery system could be exploited to target specific organs and diseases through identification of the specific heparin sulphate on the target cell.  Appropriate modifications of the targeting proteins can then be made to deliver therapeutic molecules where they are needed within the body.

This technology has advantages over other methods of intracellular delivery such as stochastic variation in expression that can occur with vectors.  Since the method is protein-based, it eliminates the risk of genomic integration and insertional mutagenesis inherent to DNA-based methodologies.  The images show the capability of the GET system to target specific cells, such a blood progenitors from murine embryonic stem cells(mESCs) (left) as well as the ability to target differentiated cells, such as those in an mESC culture (right).

Whilst this latest research is promising, Dr James Dixon, lead author on the paper, says “We still need much further work to test its safety, stability and activity inside the body and ensure we can get the drugs to the right place. We’ve shown this can be done in-vitro but it needs to be achieved before this technique can be trialled in patients. We are however very hopeful that this powerful technology can be used to help people as soon as possible.” Find out more about the technology in the associated press release by the University of Nottingham.

The technology has been licensed to a University of Nottingham spin-out company, Locate Therapeutics, Ltd to assist the translation of this research to the clinic.