Genome editing techniques for blood stem cells just got better, thanks to a collaborating team of researchers at the University of Southern California and Sangamo BioSciences. In a study published online on November 9, 2015 in Nature Biotechnology, co-first authors Colin M. Exline, Ph.D., from USC, and Jianbin Wang, Ph.D., from Sangamo BioSciences, describe a new, more efficient way to edit genes in blood-forming or "hematopoietic" stem and progenitor cells (HSPCs). The article is titled “Homology-Driven Genome Editing in Hematopoietic Stem and Progenitor Cells Using ZFN mRNA and AAV6 Donors.” "Gene therapy using HSPCs has enormous potential for treating HIV and other diseases of the blood and immune systems," said co-corresponding author Paula Cannon, Ph.D., Professor of Molecular Microbiology and Immunology, Pediatrics, Biochemistry and Molecular Biology, and Stem Cell Biology and Regenerative Medicine at USC. "And using genome editing techniques now allows us to make very precise changes that could repair genetic mutations -- the gene typos -- that can cause disease." Despite the enormous potential of such targeted gene medicine to cure patients, getting genome editing to work has proven challenging in human HSPCs -- especially in the most primitive, least differentiated cells with the greatest ability to become any blood cell type. Dr. Cannon's group, working with a team at Sangamo, has been using "genetic scissors" called zinc finger nucleases (ZFNs) to cut a cell's DNA at a precise location or sequence. The cell normally uses a copy of the cut DNA sequence as a template to repair the DNA break. During this process, there is the opportunity to introduce new DNA sequences or to repair mutations, effectively fooling the cell into making a genetic edit.
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