Gene therapy and gene editing strategies for hemoglobinopathies.

Abstract excerpt

Gene therapy for hemoglobinopathies is currently based on transplantation of autologous hematopoietic stem cells genetically modified with an integrating lentiviral vector expressing a globin gene under the control of globin transcriptional regulatory elements. Studies and safety works demonstrated the potential therapeutic efficacy and safety of this approach, providing the rationale for clinical translation....

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Combined Modality Therapy
• Share to Gene Editing
• Share to Anemia, Sickle Cell