Scientists from the University of Cambridge in the UK conducted the first treatment trial of a new type of gene therapy based on CRISPR. 10 patients suffering from the rare inflammatory disease hereditary angioedema received treatment.
The results showed that nine of them were almost completely cured. The new treatment cuts the kallikrein gene by encapsulating genetic material inside lipid nanoparticles, which are absorbed by liver cells to work. It is very attractive that patients can achieve such good results after taking it only once.