How is Genetic Therapy Advancing?

Genetic therapy, an experimental technique that involves altering genes to prevent and treat diseases, is continually advancing. New innovations are empowering scientists, researchers and doctors with the scope to address a wide range of diseases, including cancer, diabetes and AIDS. Below, we spotlight some of the most recent advances in genetic therapy and the implications for modern healthcare.

COVID-19 and mRNA technology

The COVID-19 pandemic has thrust gene therapy onto the global stage, with vaccines from Moderna and Pfizer-BioNTech using mRNA technology to combat the SARS-CoV-2 virus. Engineered to protect the body from infectious diseases, mRNA vaccines teach cells how to create proteins that trigger an immune response.

“With the success of the 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 game changer in the delivery of gene therapies” says Sally-Ann Cryan, a Professor of Drug Delivery at the Royal College of Surgeons in Ireland.

Genetic switching for biochemicals

Genetic therapy isn’t just used in the healthcare sector, with the chemical production industry also harnessing the huge potential. At the Integrative Synthetic Biology Centre in Warwick, Dr Ahmad Mannan and his team are experimenting with ways to permanently switch bacteria into chemical production mode using genetic therapy technology.

Symmetrical DNA

A recent breakthrough made at the University of Birmingham revealed that single-celled bacteria have symmetrical DNA sequences that can be read in both directions. Professor David Grainger says the discovery is “incredibly significant” and could revolutionise a range of biotechnology fields, including drug development.

Understanding genetic diversity and evolution

When it comes to treating cancers using genetic therapies, a deep understanding of the diversity and evolution pathways of tumours is fundamental. Insight into how cancers evolve is an important part of helping doctors make informed treatment decision.

“Cancers are constantly changing over time, so it’s important to recognise that a sample taken from a tumour reflects a single point in time and the cancer will continue to evolve after this,” asserts Peter Van Loo, group leader of the Cancer Genomics Laboratory at the Francis Crick Institute in London. “Understanding more about the evolution of subclones, why they develop in one direction over another, as well as how common they are, could help doctors better predict the levels of and types of variation likely to be present in a specific cancer type.”

The Francis Crick Institute has also been awarded a grant to fund the construction of a genetic map for Parkinson’s disease. Co-led by Sonia Gandhi, the project aims to chart the genetic and biological factors that drive the progressive nervous system disorder. Find out more about the multimillion pound project in ‘Building a Genetic Map for Parkinson's