AMC success in human gene therapy

Researchers at Amsterdam University’s teaching hospital AMC have carried out one of the first successful experiments in gene therapy on humans, reports the Volkskrant newspaper on Friday.


The researchers used a virus to introduce an active gene into the cells of eight patients with a rare genetic disease that hinders the creation of lipoprotein-lipase, a protein that mediates the processing of fats by the body.
By introducing the gene into their cells, the researchers were able to stimulate the patients’ muscle cells to produce the lacking protein. The technique promises to have applications in the treatment of diabetes, obesity, heart- and vascular disease, the paper says.