Gene Therapy and Genetic Cardiomyopathies

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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

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Categoria
Cardiology
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