RESEARCH & INNOVATION RECENT HIGHLIGHTS
Exciting research at RCSI reflects the contribution scientists make to healthcare and society GENE DELIVERY
S c ient i st s have de velope d polypeptide-based materials that act as effective vectors for delivering gene therapies. The platform, which is the first of its kind, enables the vectors to be adapted to suit the specific gene therapy cargo. Professor Sally-Ann Cryan The work, led by researchers from RCSI University of Medicine and Health Sciences is funded by Science Foundation Ireland (SFI). “With the success of COVID-19 vaccines, the potential of gene therapies is becoming apparent, and advanced nanoparticle delivery systems are key to enabling their use clinically. We have shown that these nanoparticles have real potential to be a gamechanger in the delivery of gene therapies,” said Professor Sally-Ann Cryan, the study’s senior author and Professor of Drug Delivery, RCSI. A major challenge for gene therapies is preparing them in a way that can deliver the genetic information into the host cells.
BLOOD CLOTTING AND LONG COVID
A new study of 50 patients, led by researchers from RCSI and published in the Journal of Thrombosis and Haemostasis, shows that patients with Long COVID syndrome continue to have higher measures of blood clotting, which may help explain persistent symptoms, such as reduced physical fitness and fatigue. Previous work by the same group studied the dangerous clotting observed in patients with severe acute COVID-19. They discovered that clotting markers were significantly elevated in the blood of patients with Long COVID syndrome compared with healthy controls. “Our results suggest that the clotting system may be involved in the root cause of Long COVID syndrome,” said Dr Helen Fogarty,
The researchers developed a platform that produces bespoke star-shaped polypeptide nanoparticles, which effectively deliver a range of therapies, including gene therapies. Crucially, these polypeptides are more flexible and easier to handle than lipids. To demonstrate the potential of this material, the researchers used it to deliver a gene therapy that regenerated bone. In preclinical work, the researchers loaded the material with DNA molecules that promote bones and blood vessels to regrow. They placed these nanomedicines in a scaffold that could be implanted into a defect site and deliver the genetic cargo into infiltrating host cells. The geneloaded scaffold accelerated bone tissue regeneration, with a six-fold increase in new bone formation compared to a scaffold alone. “While more testing is needed before these therapies can be used clinically, our platform allows us to design our polypeptides to meet a variety of delivery scenarios and provide tailored solutions to gene delivery challenges,” said Professor Andreas Heise, project collaborator and Professor of Polymer Chemistry, RCSI. “We are developing this patent-protected technology towards commercialisation, with support from an Enterprise Ireland Commercialisation Fund Award, and are seeking expressions of interest from industry partners and investors.”
the study’s lead author, ICAT Fellow and PhD student at the Irish Centre for Vascular Biology in the RCSI School of Pharmacy and Biomolecular Sciences. “Millions of people are already dealing with the symptoms of Long COVID syndrome. It is imperative that we continue to study this condition and develop effective treatments,” said Professor James O’Donnell, Director of the Irish Centre for Vascular Biology, RCSI and Consultant Haematologist in the National Coagulation Centre in St James’s Hospital, Dublin. The study was funded by the Wellcome Trust, the Health Research Board, the Irish Clinical Academic Training programme and the Irish COVID-19 Vasculopathy Study and was supported by a philanthropic grant from the 3M Foundation.
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