Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

Abstract excerpt

Sickle cell disease (SCD) is due to a mutation in the β-globin gene causing production of the toxic sickle hemoglobin (HbS; α 2 β S 2 ). Transplantation of autologous hematopoietic stem and progenitor cells (HSPCs) transduced with lentiviral vectors (LVs) expressing an anti-sickling β-globin (βAS) is a promising treatment; however, it is only partially effective, and patients still present elevated HbS levels....

Topics

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

• Share to Hemoglobinopathies and Related Disorders
• Share to Parvovirus B19 Infection Studies
• Share to Virus-based gene therapy research