Research ArticleGene Therapy

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

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Science Translational Medicine  11 Oct 2017:
Vol. 9, Issue 411, eaan0820
DOI: 10.1126/scitranslmed.aan0820

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Gene correction, one step at a time

Although gene therapy has been proposed for a variety of genetic disorders, including severe combined immunodeficiency, it has not yet found routine use in the clinic, in part because of potential complications. To help pave the way for safer translation of such gene therapy, Schiroli et al. studied potential approaches to it in mouse models of severe combined immunodeficiency. The authors systematically analyzed the outcomes of using different approaches to conditioning, different numbers of gene-edited cells, different techniques for editing the faulty gene, and other aspects of the technology to find the safest and most effective method.