Gene Therapy in a Patient with Sickle Cell Disease: Brief Report

@article{Ribeil2017GeneTI,
  title={Gene Therapy in a Patient with Sickle Cell Disease: Brief Report},
  author={J. Ribeil and S. Hacein-Bey-Abina and E. Payen and A. Magnani and M. Semeraro and Elisa Magrin and L. Caccavelli and B. Neven and P. Bourget and W. El Nemer and P. Bartolucci and Leslie Weber and H. Puy and J. Meritet and D. Gr{\'e}vent and Y. Beuzard and S. Chrétien and T. Lefebvre and R. W. Ross and O. Negre and G. Veres and L. Sandler and Sandeep Soni and M. de Montalembert and S. Blanche and P. Leboulch and M. Cavazzana},
  journal={The New England Journal of Medicine},
  year={2017},
  volume={376},
  pages={848–855}
}
Abstract Sickle cell disease results from a homozygous missense mutation in the β‐globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achieving effective, persistent inhibition of polymerization of hemoglobin S. We describe our first patient treated with lentiviral vector–mediated addition of an antisickling β‐globin gene into autologous hematopoietic stem cells. Adverse… Expand
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TLDR
Research on sickle cell anemia has again taken center stage because of new drug therapies, cures through stem cell transplantation, and the promise of gene therapy. Expand
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TLDR
The potential and the challenges of gene addition and gene editing strategies for the hemoglobin diseases are discussed and a relevant chance to this group of patients for whom cure has previously not been on the horizon is offered. Expand
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TLDR
In follow-up multicenter Phase II trials with an essentially identical vector (termed LentiGlobin BB305) and protocol, 12 of the 13 patients with a non-β0/β0 genotype, representing more than half of all transfusion-dependent β-thalassemia cases worldwide, stopped red blood cell transfusions with total hemoglobin levels in blood approaching normal values. Expand
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.
Sickle cell disease (SCD) is caused by a mutation in the β-globin gene leading to polymerization of the sickle hemoglobin (HbS) and deformation of red blood cells. Autologous transplantation of… Expand
Fetal Hemoglobin in Sickle Cell Anemia.
TLDR
A confluence of detailed understanding of the molecular basis of HbF gene expression, coupled with the ability to precisely target by genomic editing most areas of the genome, are producing important preliminary therapeutic results that could provide new options for cell-based therapeutics with curative intent. Expand
Genetic Therapies for Sickle Cell Disease.
TLDR
This review focuses on the current state of the field, factors that determine clinical success, gene editing, and future prospects, and significant progress has been made to clinically translate gene therapy for sickle cell disease using lentivirus vectors carrying antisickling genes. Expand
Gene therapy for sickle cell disease.
TLDR
This systematic review has identified the need for well-designed, randomised controlled trials to assess the benefits and risks of gene therapy for sickle cell disease and concluded no objective conclusions or recommendations in practice can be made. Expand
MOLECULAR MEDICINE: Found in Translation.
TLDR
How the promise of molecular medicine to bring transformative treatments to the clinical arena is finally being realized is reviewed. Expand
Gene therapy for sickle cell disease.
TLDR
A systematic review has identified the need for well-designed, randomised controlled trials to assess the benefits and risks of gene therapy for sickle cell disease and concluded no objective conclusions or recommendations in practice can be made. Expand
Sickle cell disease: progress towards combination drug therapy
TLDR
Progress made towards drug development and potential combination drug therapy for SCD with the standard of care hydroxycarbamide is discussed. Expand
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