• Date: November 10, 2020

 

On 7 October, Emmanuelle Charpentier and Jennifer Doudna won the Nobel Prize in Chemistry "for the development of a method for genome editing", commonly known as CRISPR-Cas9, which means that DNA can be cut at very specific points. These new “genetic scissors” are truly ground-breaking in the fields of biology and medicine. Among other things, they have the potential to develop new therapies to treat cancer and cure hereditary genetic diseases.

However, this genome editing tool is not the only one. In fact, the Spanish team at the Translational Synthetic Biology Research group at Pompeu Fabra University (UPF) is developing a new piece of technology called Uni-large, which could be even more successful than the award-winning CRISPR-Cas9, since it is more universal, effective and safe. In this article, we interviewed Marc Güell, the Head of the Translational Synthetic Biology group at UPF, who is running this project. Uni-large receives funding from CaixaImpulse, a "la Caixa" Foundation Research and Health project, which promotes the transfer of research to society. Below, Güell tells us some of the secrets behind this promising technique.

Uni-large, promising “genetic scissors”

First things first, what is Uni-large?

Uni-large is a new piece of genome editing technology that allows us to cut and paste DNA precisely and effectively, and it also allows us to paste pieces of DNA that are very long. It’s almost like “genetic scissors”, which is what genome editing tools are commonly known as.

What can we achieve with these “genetic scissors”?

Genome editing tools have an infinite number of applications in different fields of bioengineering. In therapeutic terms, they allow us to reprogramme “errors” in the genetic code that lead to diseases: we can cut out a DNA sequence and replace it with another one that’s in a good condition so that it goes back to its normal function and sends the disease into remission.

What sets Uni-large apart from other genome editing tools such as CRISPR-Cas9?

Firstly, it’s more universal; while the CRISPR method corrects individual mutations in each patient, Uni-large has the potential to reach all patients that suffer from a certain disease. Furthermore, CRISPR cuts the genome and uses DNA repair in the cell to edit, which could have unpredictable results. However, we implement stricter controls on the editing through an extra element that always cuts and pastes in the same way, making it safer. Lastly, Uni-large is more effective than other “genetic scissors” when working on large genes, which could help us to cure hereditary diseases with these characteristics.

Could you give us an example?

Right now, we're using Uni-large on merosin-deficient muscular dystrophy. This disease is due to a mutation in the LAMA2 gene. Although this is the most common form of congenital muscular dystrophy, there is currently no treatment available, so our goal is to cure it.

What potential could this technique have in the future?

Based on the idea that we and other animals that are made up of the same biological constituent parts as us, we could resist cosmic radiation, or basically become resistant to cancer, or even live forever—engineering in biological systems really has unlimited properties. All in all, working on DNA, the true language of life, doesn’t just have huge potential, but also many advantages.

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