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From Viruses to Lipid Nanoparticles: A Focus on Non-Viral Integrating Gene Therapy for Classical Hematologic Disorders

Juliana Perez Botero, MD

.... Valoctocogene roxaparvovecv received approval in Europe to treat hemophilia A. In the United States, the U.S. Food and Drug Administration authorized beti-cel (betibeglogene autotemcel) for patients with β-thalassemia, and etranacogene dezaparvovec for the treatment of adults with hemophilia B. The first...)

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Optimism Versus Reality

Patrick Ellsworth, MD

..., they in theory lend themselves quite well to a genetic cure. At this year’s joint session, we have the chance to hear from a handful of the world’s experts in the field of gene editing, with the topics substantively addressing hemoglobinopathies such as sickle cell disease and thalassemia, as well as other blood...)

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From Viruses to Lipid Nanoparticles: A Focus on Non-Viral Integrating Gene Therapy for Classical Hematologic Disorders

Optimism Versus Reality

ASH Publications

American Society of Hematology

From Viruses to Lipid Nanoparticles: A Focus on Non-Viral Integrating Gene Therapy for Classical Hematologic Disorders

Optimism Versus Reality

ASH Publications

American Society of Hematology

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