Educational content on VJHemOnc is intended for healthcare professionals only. By visiting this website and accessing this information you confirm that you are a healthcare professional.

Share this video  

BSH 2021 | Gene therapies in sickle cell

David Rees • 27 Apr 2021
BSH 2021
Myelodysplastic Syndromes Non-Malignant Sickle Cell Disease

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.

More from David Rees

1:23
Emerging drug targets for sickle cell disease
David Rees • 27 Oct 2023
1:36
Determinants of severity in sickle cell disease
David Rees • 27 Oct 2023
2:19
The Lancet Commission on sickle cell disease
David Rees • 27 Oct 2023
2:05
Biomarkers in sickle cell disease
David Rees • 27 Apr 2021

Related Videos

2:01
The MDT in the new era of gene therapy for hemoglobinopathies
Mattia Algeri • 15 Apr 2024
2:22
Haplo-SCT for older patients with AML/MDS, and should age be a barrier to transplantation?
Raynier Devillier • 15 Apr 2024
2:31
Emerging treatment options for patients with ESA-refractory MDS: luspatercept & imetelstat
Eva Hellström-Lindberg • 11 Apr 2024
1:49
The unmet needs of patients with VEXAS syndrome
Maël Heiblig • 17 Apr 2024
The content of VJHemOnc is intended for healthcare professionals