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Sickle Cell Disease and Gene Therapy — Patient and Physician Perspectives | NEJM

In this short documentary video from the New England Journal of Medicine (NEJM), patients and physicians partner both to highlight the experience of living with sickle cell disease and to discuss the pathophysiology of the disease and new treatment strategies, including gene therapy. Patients share their own stories of their interactions with the health care system and explore the challenging topics of racial disparity and health equity. Physicians express a cautious optimism as they review the risks and benefits of gene therapy. The New England Journal of Medicine is the world’s leading general medical journal. Continuously published for over 200 years, NEJM publishes peer-reviewed research along with interactive clinical content for physicians, educators, and the global medical community at https://www.NEJM.org. 0:00 Impact on Patients 2:20 Pathophysiology, Gene Therapy, and Disease Burden 5:38 Health Equity and History of Treatment 8:20 Gene Therapy Risk-Benefit #sicklecelldisease