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Curative Strategies for Sickle Cell Disease: The Future of Gene Editing
Manage episode 509554149 series 3693262
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Guest: Alexis Leonard
With two FDA-approved gene therapies, including the first CRISPR-based treatment, curative strategies for sickle cell disease are continuing to expand the treatment landscape. However, challenges remain in optimizing safety and accessibility, particularly for patients unable to tolerate current myeloablative conditioning. Learn about advancements in ex vivo editing, such as improved stem cell targeting and multiplex editing, and the possibility of in vivo approaches that may help transform sickle cell disease management with Dr. Alexis Leonard, who works in the Department of Hematology at St. Jude’s Hospital in Memphis, Tennessee.
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With two FDA-approved gene therapies, including the first CRISPR-based treatment, curative strategies for sickle cell disease are continuing to expand the treatment landscape. However, challenges remain in optimizing safety and accessibility, particularly for patients unable to tolerate current myeloablative conditioning. Learn about advancements in ex vivo editing, such as improved stem cell targeting and multiplex editing, and the possibility of in vivo approaches that may help transform sickle cell disease management with Dr. Alexis Leonard, who works in the Department of Hematology at St. Jude’s Hospital in Memphis, Tennessee.
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