Gene Therapy and Classical Homocystinuria

Gene Therapy and Classical Homocystinuria

Our Scientific Advisory Board’s co-chair, Professor Warren Kruger is pioneering gene therapy for classical homocystinuria. Prof. Kruger joins the Journal of Inherited Metabolic Disease Podcast to talk about homocystinuria, successful trials in gene therapy, why it costs so much to make viruses and what inspires him. Take a listen to the podcast here. You can also read Prof. Kruger’s publication ‘Long-term functional correction of cystathionine β-synthase deficiency in mice by adeno-associated viral gene therapy’ here.