Spain, Medicine, Genetics
Gene correction for a rare disease Angeles suffers from a severe and rare genetic disease called Acute Intermittent Porphyria (AIP). This means, one of her genes restrains her liver to produce a specific protein needed for the metabolism of the blood.
Renewed hope for gene therapy in rare disease Between 30 and 40 million people in Europe suffer from rare diseases —many of them children. As most of these diseases have genetic origins, gene therapy is a major hope for their future cure .