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 decade old, clinical development has been hampered by poor efficiency of gene transfer in viral-mediated gene therapies, leading to low yields...