We recently collaborated with doctors at the Centre of Regenerative Medicine, Università di Modena e Reggio Emilia and University Children’s Hospital of Bochum who saved a ‘butterfly child’ from the life-threatening effects of a rare genetic disease (known as epidermolysis bullosa) by transplanting new skin derived from his genetically modified stem cells. Prior to transplanting, IGA researchers have analyzed the regenerated skin cells using NGS technology and advanced bioinformatic tools to detect the integration sites of the retroviral vectors – used for delivering the gene therapy – in the genomes of the stem cells that produced the life-saving new skin.
The scientific report of this work has been published in Nature. Click here for media coverage at BBC, CNN and NYTimes.