Fernando Civeira Murillo
Professor of Medicine and Head of the Lipid Research Group at the University of Zaragoza
Does the press release accurately reflect the study?
‘I think the press release is very good and accurately reflects the problem and the solution provided by the paper’.
Is the study of good quality?
‘The study is excellent and only has the limitation that the follow-up is short, only a few months, and we will have to wait to see how the child evolves.
How does this work fit in with the existing evidence?
‘Perfectly, it is the future. Gene editing à la carte, individualised for each pathogenic variant responsible for the disease.
Are there any major limitations to be taken into account?
‘The participating centres are world leaders in the technology. The limitation is time, as a severe disease that does not allow for delays in doing further animal testing and experimentation with the therapy’.
What are the implications for the real world?
‘Huge. Probably in a few years the vast majority of severe monogenic diseases will disappear thanks to gene editing’.