Journal
HUMAN GENE THERAPY
Volume 23, Issue 7, Pages 668-675Publisher
MARY ANN LIEBERT, INC
DOI: 10.1089/hum.2012.116
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Funding
- Wellcome Trust
- GOSH Children's Charity
- Medical Research council
- European Commission FP7 CELL-PID
- Great Ormond Street Hospital Childrens Charity [V1242, V1259, V1223] Funding Source: researchfish
- Medical Research Council [G0501969, G0802483, G0600773] Funding Source: researchfish
- National Institute for Health Research [NF-SI-0611-10001] Funding Source: researchfish
- MRC [G0501969, G0802483, G0600773] Funding Source: UKRI
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For over 40 years, primary immunodeficiencies (PIDs) have featured prominently in the development and refinement of human allogeneic hematopoietic stem cell transplantation. More recently, ex vivo somatic gene therapy using autologous cells has provided remarkable evidence of clinical efficacy in patients without HLA-matched stem cell donors and in whom toxicity of allogeneic procedures is likely to be high. Together with improved preclinical models, a wealth of information has accumulated that has allowed development of safer, more sophisticated technologies and protocols that are applicable to a much broader range of diseases. In this review we summarize the status of these gene therapy trials and discuss the emerging application of similar strategies to other PIDs.
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