Gene therapy is changing how clinicians care for patients with rare and genetic diseases, with more therapies moving from research into clinical use. In this episode, Dr. Nirali Shah is joined by Dr. Christy Duncan and Dr. Sung‑Yun Pai to talk through recent progress in the field and what it actually takes to deliver these treatments at the patient level.
The discussion draws on their experience in transplant and genetic disease, covering both scientific advances and the operational challenges that come with implementing gene therapy — especially in complex and resource‑intensive settings.
Tune in for a conversation that covers:
Recent developments in gene therapy, including in vivo approaches and newly approved treatments
Barriers to broader use, including access, equity, and long-term monitoring
What centers need to consider when building a gene therapy program, from workflows to team coordination
Where things may be heading, including more individualized therapies and efforts to expand availability
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