The Myelodysplastic Syndromes Channel is supported with funding from Geron (Silver).
The Non-Malignant Channel is supported with funding from Agios (Gold).
The Sickle Cell Disease Channel is supported with funding from Agios (Gold).
VJHemOnc is an independent medical education platform. Supporters, including channel supporters, have no influence over the production of content. The levels of sponsorship listed are reflective of the amount of funding given to support the channel.
BSH 2021 | Gene therapies in sickle cell
David Rees, MA, MBBS, FRCP, FRCPath, FRCPCH, King’s College Hospital, London, UK, discusses the future of gene therapies in treating sickle cell disease (SCD). Prof. Rees outlines the findings of a trial (NCT04293185) evaluating gene therapy with a lentiviral vector, which was stopped due to development of myelodysplastic syndrome (MDS) in patients. Additional approaches involve attempts to increase fetal hemoglobin (HbF) levels or knockdown inflammatory markers, but these approaches fail to completely cure SCD. Future developments may involve in vivo gene therapies that can correct the sickle cell mutation. This interview took place at the 2021 British Society for Haematology Annual Scientific Meeting.
