A study led by researchers from the Royal College of Surgeons in Ireland (RCSI) presented a new adaptable nanoparticle platform that enables enhanced delivery of various therapies, including gene therapy.

Science Codex reported that researchers had developed polypeptide-based materials that can be vectors for delivering gene therapies. They noted that this is the first-of-its-kind platform that allows the vectors to be adapted to suit the specific gene therapy cargo.

The study, entitled "Gene activated scaffolds incorporating star-shaped polypeptide-pDNA nanomedicines accelerate bone tissue regeneration in vivo," was published in Biomaterials Science. It was led by researchers from RCSI University of Medicine and Health Sciences and funded by Science Foundation Ireland.


 Polypeptide Nanoparticle Designed to Adapt to Gene Therapy Cargo For A More Efficient Delivery System
(Photo: Wikimedia Commons)
Gene therapy

What is Gene Therapy?

According to Medline Plus, gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, doctors hope to use this technique to insert genes into the cells of patients instead of using drugs or surgery.

Indeed, it is a promising treatment that doctors hope to use someday. But it still needs more research to ensure that it is safe and effective to use. Gene therapy is currently being tested for diseases without any cure.

ALSO READ: Novel Gene Therapy Seen to Preserve Memory, Learning Functions in Alzheimer's Disease Patients


Testing Polypeptide Nanoparticles for Gene Therapy

RCSI researchers said that one of the major challenges in gene therapies is preparing them to deliver the genes to the host cells. The team tested the newly developed platform of the star-shaped polypeptide nanoparticles, which could effectively deliver various therapies like gene therapy.

These nanoparticles are more adaptable and easier to handle than lipid nanoparticles used in delivering and maintaining the mRNA technology of COVID-19 vaccines.

They used the nanoparticles to deliver gene therapy that regenerated bone. According to Drug Target Review, the team loaded the material with DNA that promotes bone and blood vessel regrowth during the pre-clinical work.

They put the nanomedicines in a scaffold which is then implanted into a defective site of the bone where the genetic cargo is delivered and will infiltrate the host cells.

Researchers found that the gene-loaded scaffold hastens the regeneration of bone tissue of about six times regrowth in bone formation compared to the scaffold alone.

Nanoparticles: A Game-Changer in Delivery of Gene Therapies

Study senior author professor Sally-Ann Cryan, a professor of Drug Delivery at RCS, said that the success of COVID-19 vaccines showed the potential of gene therapies.

More so, it also reveals that nanoparticle delivery systems play a crucial role in the delivery of gene therapies. She added that the new platform enables them to design polypeptides that adapt to the gene therapy cargo and provide solutions to gene delivery challenges.

"We are developing this patent-protected technology towards commercialization, with support from an Enterprise Ireland Commercialisation Fund Award, and are seeking expressions of interest from industry partners and investors," she said according to Science Codex.

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