NEW TECHNOLOGIES FOR GENETIC DISEASES

Giulia Bortolussi, researcher ICGEB

Nicola Brunetti-Pierri, principal investigator Telethon Institute of Genetics and Medicine, professor of Pediatrics at Federico II University of Naples

Federico Mingozzi, Chief Executive Officer Newco

Andrés Muro, group leader Mouse Molecular Genetics Laboratory ICGEB

Laura Celotto, communications office ICGEB

Treating genetic diseases by correcting the gene mutations that cause them is one of the most promising challenges in medicine today. This includes approaches like the use of viral vectors and gene-editing tools such as CRISPR/Cas9. How do basic and translational research contribute to the application of these therapies in clinical practice? Which approach is better suited for personalized medicine? We'll discuss these questions with the authors of the first clinical demonstration of the effectiveness of a gene therapy for the extremely rare Crigler-Najjar syndrome, a liver disease, published in the New England Journal of Medicine.