First successful gene therapy in adults
An international team of researchers has achieved the world's first successful gene therapy in adults. Two patients with septic granulomatosis, a congenital immune deficiency, have been treated at the Frankfurt University Hospital in the past two years. This was announced by the treatment coordinator, Dorothee von Laer, from the Georg-Speyer-Haus in Frankfurt.
Blood stem cells were taken from the two 25 and 26-year-old patients, into which the research team then introduced he althy copies of the gene gp91phox. The gene, which is incorrectly mutated in the X-linked inherited granulomatosis, encodes an immune protein of the phagocytes, a subgroup of the defense cells of the immune system. In patients with the disease, the failure of the gene leads to a limited ability of the body's defenses to take oxidative action against microbial germs.
The gene-treated patients then had the modified stem cells transplanted back. As a result, the proportion of he althy phagocytes rose to more than fifty percent, infections regressed and serious new diseases did not occur.
Gene therapy is a hope especially for seriously ill people for whom no bone marrow donor can be found, says von Laer. However, it cannot yet be completely ruled out that patients will develop blood cancer as a result of gene therapy. It is currently being researched how this possible side effect can be prevented. The new technology could also potentially be used to treat a wide range of rare inherited diseases in patients who have a defective gene.