4:12 PM
Oct 11, 2017
 |  BC Extra  |  Preclinical News

Researchers optimize gene editing for SCID

Researchers from Ospedale San Raffaele and elsewhere used zinc finger nuclease and CRISPR-Cas9 (CRISPR-associated protein 9)-based gene editing to correct hematopoietic stem/progenitor cells in animal models of SCID-X1, demonstrating improved gene editing protocols that could treat the disease. The results were published in Science Translational Medicine.

Although the strategy of gene correction in patient-derived hematopoietic stem/progenitor cells (HSPCs) for treating X-linked severe combined immunodeficiency (SCID-X1) is more than a...

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