Gene therapy for monogenic disorders of the bone marrow

@article{Ghosh2015GeneTF,
  title={Gene therapy for monogenic disorders of the bone marrow},
  author={Sujal Ghosh and Adrian J. Thrasher and H Bobby Gaspar},
  journal={British Journal of Haematology},
  year={2015},
  volume={171}
}
Ex‐vivo gene transfer of autologous haematopoietic stem cells in patients with monogenic diseases of the bone marrow has emerged as a new therapeutic approach, mainly in patients lacking a suitable donor for transplant. The encouraging results of initial clinical trials of gene therapy for primary immunodeficiencies were tempered by the occurrence of genotoxicity in a number of patients. Over the last decade, safer viral vectors have been developed to overcome the risk of insertional… 

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