A promising new version of a CRISPR-based gene therapy has shown potential in curing a rare genetic condition called hereditary angioedema. This condition causes individuals to experience sudden episodes of tissue swelling that can be life-threatening. The therapy involves making cuts in the gene for kallikrein, a protein involved in inflammation.
In a trial of ten individuals with hereditary angioedema, nine experienced a significant reduction in swelling attacks after receiving the gene treatment. Over the course of six months, their attacks decreased by 95%. Additionally, all but one participant remained free of further episodes for at least a year, indicating the possibility of a cure.
Hereditary angioedema is caused by mutations in a gene that produces a protein called C1-inhibitor, which helps regulate inflammation. Existing medications for the condition work by blocking a different molecule involved in inflammation called kallikrein. The new therapy, developed by Intellia Therapeutics, directly targets the kallikrein gene using lipid nanoparticles that are taken up by liver cells.
One notable aspect of this treatment is that it is administered directly into the body, known as "in vivo" delivery. This method is more straightforward and attractive compared to the more complex and time-consuming "ex vivo" approach, which involves removing cells from the body, modifying them in the lab, and reintroducing them.
CRISPR gene therapies are being explored for various genetic conditions, with the UK and the US recently approving the first treatment for sickle cell disease and beta-thalassemia. The success of the hereditary angioedema trial is seen as an exciting development, as it opens up possibilities for treating other conditions caused by mutated proteins produced in the liver.
Overall, this new CRISPR-based gene therapy shows promise in providing a potential cure for hereditary angioedema. Further research and trials are needed to confirm its effectiveness and safety, but the initial results are encouraging. The success of this approach highlights the potential of gene therapies in addressing various genetic conditions and offers hope for future treatments.