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Genetic Therapies in Sickle Cell Disease



Advances in our understanding of genetic therapies is helping researchers find ways to potentially change, or alter, the genes that cause diseases, such as sickle cell disease. This webinar provides an overview of genetic therapies–what they are, how they work, and how genetic therapies are being explored to prevent or treat diseases. The webinar also highlights activities of the Cure Sickle Cell Initiative, an NHLBI-led collaborative research effort to accelerate the development of genetic therapies to cure sickle cell disease, and feature a patient’s perspective on participating in clinical trials and the importance of safe and widely available cures.

The webinar speakers are experts in sickle cell disease and one is a patient who participated in a sickle cell clinical study:

• Traci Mondoro, Ph.D., Chief of Translational Blood Science and Resources Branch, Division of Blood Diseases and Resources (DBDR), NHLBI
• Mark Walters, M.D., Professor of Pediatrics/Jordan Family Director, BMT Program, Interim Director of Research, UCSF Benioff Children’s Hospital, Oakland
• Lynndrick Holmes, patient representative

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