Could Lentivirus Overcome the AAV Gene-Therapy Challenges in Hemophilia A?

Despite the unprecedented evolution of new replacement and nonreplacement therapies, many unmet needs remain with respect to the treatment of severe hemophilia A.1 These unmet needs include preventing factor VIII–induced immunogenicity, providing consistent hemostatic protection during administration of replacement therapies, ensuring lifelong administration, and preventing musculoskeletal deterioration that…

​   The New England Journal of Medicine: Search Results in Hematology/Oncology