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14 October 2016

The authors explain that gene editing technology allows for targeted repair of disease-causing mutations. They are led by Dr Kyung-Rok Yu of the National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland, USA.

 
Gene editing technology has the potential to be applied to haematopoietic stem cells, offering hope of curing many hereditary and congenital diseases. However, the authors point out: "This young field still faces many challenges before it is ready for human testing."
 
In their article published in Human Gene Therapy recently, the team outline the obstacles they have overcome in the development of gene editing approaches.
 
Journal editor, Dr Terence Flotte, writes: "Gene editing is the hottest new technology in gene therapy. The use of this approach to genetically modify haematopoietic stem and progenitor cells is very promising, but requires a careful assessment."
 
After almost two decades of intensive research, clinical trials treating various inherited immune deficiency diseases, including X-linked severe combined immunodeficiency, with haematopoietic stem and progenitor cell gene therapy are yielding promising results.
 
But the drawbacks include a limited capacity to insert transgenes into certain haematopoietic stem and progenitor cells, and the risk of genotoxicity. This has led to the development of viral vectors that are safer, but remain effective.
 
The authors hope that ultimately, "given their unique properties", a small number of genetically modified haematopoietic stem and progenitor cells "could accomplish lifelong, corrective reconstitution of the entire hematopoietic system in patients with various haematologic disorders".
 
Kyung-Rok, Y. et al. Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles. Human Gene Therapy 2 August 2016 doi:10.1089/hum.2016.107
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