CRISPR

The European Medicines Agency (EMA) has recommended approval of the first drug in the European Union to use the CRISPR/Cas9 gene-editing technique. The drug, Casgevy, is indicated for the treatment of two rare inherited diseases, beta thalassaemia and sickle cell disease (sickle cell anaemia), caused by genetic mutations that affect the production or function of haemoglobin, the oxygen-carrying protein in red blood cells. Both conditions are debilitating and potentially fatal. The EMA opinion will be sent to the European Commission for a decision on an EU-wide marketing authorisation.

A team of scientists led by the Catholic University of America in Washington has designed new artificial vectors based on viruses to improve gene therapy processes. The main novelty is that they are constructed from viruses that infect bacteria. Among other advantages, this would make it possible to avoid the possible memory of our defences against them and have a greater capacity. According to the authors, who publish their results in the journal Nature Communications, these nanoparticles "have the potential to transform gene therapies and personalised medicine".

In January 2013, two laboratories demonstrated that CRISPR tools could be used to edit genes in human cells. Ten years later, the first patients are already benefiting from the molecular scissors to overcome incurable diseases. This week in Science, one of the pioneers of CRISPR, Nobel laureate Jennifer Doudna, summarises the history of these tools, without forgetting that it all began thirty years ago with the findings of Francis Mojica in the Santa Pola salt flats.

Ischaemia-reperfusion injury is one of the causes of damage caused by diseases such as myocardial infarction. A study published in the journal Science has used base editors, a gene-editing tool derived from CRISPR, to modify a key protein in the hearts of mice. According to the authors, the intervention allowed them to recover their function after a heart attack and could potentially be used in a wide range of patients, as it does not depend on the presence of a specific mutation.

An international study led by Spanish researchers has succeeded in reconstructing the ancestors of the CRISPR-Cas system present in extinct bacteria from up to 2.6 billion years ago. The reconstructed systems work and are more flexible than the current ones. According to the authors, this could open up new avenues for gene editing. The results are published in the journal Nature Microbiology.

The Spanish Confederation of Scientific Societies (COSCE) has just published a report, submitted to the EU, which proposes and compares different possibilities for reform of the regulatory framework for genome editing techniques.