Previously, scientists have extracted human immune cells, then edited the genes in them and put them back as a means of giving those cells the ability to attack lung cancer.
If Madeux’s treatment is successful, it would be the first time a gene therapy has worked from inside the human body. To achieve the gene editing, Sangamo is using a technology called zinc-finger nucleases.
Along with the zinc-finger nucleases, the virus also contains snippets of the missing gene, which is then inserted by the body’s own gene-repairing system into the place where the cut is made.
Once the gene is part of a person’s DNA, it will be with them forever.
Past gene therapy trials have ended in tragedy, where although inserting genes have helped alleviate the problem, the tool also injected genes into unintended parts of the DNA, which caused harm.
It’ll also be a big boost for gene editing, opening up the potential for treating diseases that have remained incurable until now.