NEW TECHNOLOGIES FOR GENETIC DISEASES

Giulia Bortolussi, ricercatrice ICGEB

Nicola Brunetti-Pierri, principal investigator Istituto Telethon di Genetica e Medicina, docente di Pediatria Università Federico II Napoli

Federico Mingozzi, Chief Executive Officer Newco

Andrés Muro, responsabile Laboratorio di Genetica Molecolare Murina ICGEB

Laura Celotto, ufficio comunicazione 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.