New findings speed progress towards affordable gene therapy

New findings speed progress towards affordable gene therapy

In a promising advance for affordable, personalised medicine, researchers have used metal-organic frameworks to successfully deliver the genetic snipping tool CRISPR/Cas9 into human cancer cells.

Researchers used metal-organic frameworks (MOFs) enhanced with a green tea phytochemical coating to target human prostate cancer cells for the first time.

The new method for deploying the genetic snipping tool directly into target cells is a big step towards more effective, safer and cheaper gene therapy with treatment potential for multiple genetic disorders.

Lead researcher, Associate Professor Ravi Shukla, said MOFs, which are versatile and biocompatible nanomaterials, were a promising alternative to existing viral methods for delivering the gene editing tool CRISPR/Cas9.

“MOFs have the capacity to carry larger genetic loads and as a non-viral option, have the added benefit of being a safer prospect for patients than viral alternatives,” he said.

MOFs on the surface of metastasising prostate cancer cells. Image credit: ChemComm.

Awarded the 2020 Nobel Prize in Chemistry, CRISPR/Cas9, is widely acknowledged as a breakthrough in genetic editing for its ability to remove and replace defective DNA, however the biggest challenge remains effectively delivering it to cells.

There are currently just 13 approved methods in trial globally and all rely on viral therapies, an approach which is both extremely costly and has associated health risks.

RMIT has partnered with the CSIRO who have previously developed a technology to carry and protect biomolecules with MOFs.

CSIRO Research Team Leader, Dr Cara Doherty, said the CSIRO had also developed technologies to manufacture industrial scale quantities of various MOFs, with the potential to significantly reduce the cost of bringing them to market.

“We’re excited to find a novel way in which these materials can address complex biological issues, including targeting specific medicinal purposes like gene therapy,” she said.

L-R: Associate Professor Ravi Shukla and PhD researcher Arpita Poddar in their lab.

Cells are not designed to naturally take up foreign genes or DNA material, and the risks associated with introducing a virus into the body have slowed the progress of research into viral approaches to gene therapy.

To further improve the ability of the MOF to enter the host cell, it was coated with a phytochemical found in green tea, called epigallocatechin-gallate (EGCG), known for its antioxidant and anticancer properties.

Co-author Arpita Poddar said the EGCG worked by binding to the surface of the MOF, assisting it to enter the host cell.

“We found an increase in cellular uptake of more than 23% for EGCG coated MOFs compared to uncoated ones,” she said.

The latest findings build on previous work by the team who developed a proof of concept for the delivery model late last year.

Next, they will work to further test this technology for its application in targeting several other disease-causing genes.

The paper ZIF-C for Targeted RNA Interference and CRISPR/Cas9 Based Gene Editing in Prostate Cancer is published in ChemComm (DOI:10.1039/D0CC06241C).

 

Story: Grace Taylor

20 November 2020

Share

20 November 2020

Share

  • fashion
Engage

Related News

Dimitri Lévêque, Policy Officer for Academic Cooperation and Scientific Partnerships for North-East Asia, Indonesia and Oceania, Ministry for Europe and Foreign Affairs; Jean Thèves, Deputy Director European and International Affairs, CNRS; Christophe Corre, Director of Research, Ecole Centrale de Lyon; Stefan Enoch, Deputy Vice-President Science and Technologies, Aix-Marseille University; Australia’s Ambassador to France Her Excellency Gillian Bird; RMIT Vice-Chancellor and President Professor Alec Cameron, Marie Maillart, Associate Director Operations, RMIT Europe; Prisca Ramesh, Australia Desk Officer, Asia and Oceania Division, Ministry for Europe and Foreign Affairs and Stephanie Aeuckens, Counsellor, Australian Embassy in France. Credit: Australian Embassy in France, RMIT University

RMIT University to lead €15.7 million Australia-France doctoral training network

In a significant win for global research training, Australian and French academic ties are set to strengthen with today’s announcement of the Australia France Network of Doctoral Excellence (AUFRANDE).

Technology is key to transforming systemic deficiencies in Australia’s aged care sector, new report finds

RMIT-Cisco’s Health Transformation Lab launches report: 'Transforming aged care – towards a future in which digitisation drives respect and connection', in response to the Royal Commission into Aged Care Quality and Safety.

Sunscreen being applied to an arm. Sunscreens are an important part of sun protection and preventing serious sun damage to people – 2 in 3 Australians will develop skin cancer by the age of 70. Credit: Adobe Stock

Take the burn out of sunscreen testing: experts

Exposing humans to ultraviolet radiation to test sunscreen effectiveness should be phased out, according to scientists and cancer experts.

Dr Marco Capelli, one of the co-researchers, in the ARC Centre of Excellence for Nanoscale BioPhotonics laboratories at RMIT University. Photo: RMIT University

Discovery could inspire new way to detect brain abnormalities

Scientists have taken a promising step towards a new generation of accurate, affordable and portable devices to detect concussion, epilepsy and dementia.

Show more
aboriginal flag
torres strait flag

Acknowledgement of Country

RMIT University acknowledges the people of the Woi wurrung and Boon wurrung language groups of the eastern Kulin Nation on whose unceded lands we conduct the business of the University. RMIT University respectfully acknowledges their Ancestors and Elders, past and present. RMIT also acknowledges the Traditional Custodians and their Ancestors of the lands and waters across Australia where we conduct our business - Artwork 'Luwaytini' by Mark Cleaver, Palawa.

More information