Gene Therapy and Genetic Cardiomyopathies

Guest: John Giudicessi, M.D., Ph.D. Host: Malcolm Bell, M.D. In this podcast, listeners will learn what gene therapy is and the types of genes therapies under development for an array of genetically mediated cardiovascular disorders. In addition, active and upcoming gene therapy clinical trials for the treatment of specific arrhythmogenic, dilated, hypertrophic and restrictive cardiomyopathy genotypes will be discussed. Topics Discussed: • What is gene therapy? • How do gene therapies differ from conventional management of genetic arrhythmogenic, dilated, hypertrophic and restrictive cardiomyopathies? • Are there different types of gene therapies? • What gene therapy trials are currently active or upcoming at Mayo Clinic and other institutions? 00:00 Gene therapy focus and key guest 00:58 What gene therapy is 01:44 Gene therapy vs cell therapy 02:18 Which genetic heart diseases matter 03:13 How gene therapy is delivered 04:22 Using AAV vectors and heart targeting 06:55 How treatment is administered (IV) 07:24 How much of the heart is treated 08:24 Phase 1 trials: dosing and safety 08:53 HCM example and “critical mass” 10:15 One-and-done or future “top-offs” 11:55 Key risks and adverse effects 13:47 Other approaches, including gene editing 14:59 Trials to watch and referrals 16:55 Why genetic testing is essential 17:31 Wrap-up and future outlook 18:45 Mayo Clinic CV education resources From Mayo Clinic to your inbox, sign-up for free: https://mayocl.in/3e71zfi Visit Mayo Clinic: https://mayocl.in/2COVmlm Like Mayo Clinic on Facebook: https://www.facebook.com/mayoclinic/ Follow Mayo Clinic on Instagram: https://www.instagram.com/mayoclinic/ Follow Mayo Clinic on X, formerly Twitter: https://x.com/MayoClinic Follow Mayo Clinic on Threads: https://www.threads.net/@mayoclinic