Science and Technology Daily (Reporter Liu Xia) 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, suggesting that the new gene therapy may be able to cure dangerous inflammatory diseases. A related research paper was published in the New England Journal of Medicine on February 1.
Hereditary angioedema causes sudden swelling of body tissue, affecting areas such as the face or throat. The symptoms are similar to those of an allergic reaction, but they cannot be treated with allergy medications. It can be extremely painful and can lead to suffocation if the throat becomes blocked.
In the latest trial, 10 patients received a new type of gene therapy administered internally. The results showed that 6 months after treatment, their swelling episodes dropped by 95%. In the next 6 months to a year, 9 people did not experience any further swelling.
The research team said that existing drugs to treat hereditary angioedema work by blocking kallikrein, a protein produced by the liver that is related to inflammation. The new treatment cuts the kallikrein gene by encapsulating genetic material inside lipid nanoparticles, which are absorbed by liver cells to work.
To date, most CRISPR-based gene therapies are edited "in vitro" in the laboratory, where cells are first removed from the body, edited and then injected into the body, a complex and lengthy process. The new gene therapy uses direct administration to complete the treatment. It is very attractive that patients can achieve such good results after taking it only once.