- An upcoming article in Nature Biotechnology explains how researchers at the University of Southern California (USC) and Sangamo Biosciences have discovered a better, more efficient way to repair and deliver DNA to hematopoietic stem and progenitor cells (HSPCs)—a process known as gene editing.
- Gene therapy using HSPCs has the potential to treat HIV and other diseases of the blood and immune systems.
- Although this type of gene therapy is not yet available for clinical application, the researchers are confident that they are one step closer.
The advances in genome editing have largely bee driven by use of "genetic scissors" called zinc finger nucleases (ZFNs) to cut a cell's DNA at a precise location or sequence. The research collaborators involved in this process used ZFNs to target precise parts of the DNA in order to repair genetic mutations. Then they used viral vectors to deliver the revised DNA without disrupting the HSPCs.
By combining these two delivery methods, the scientists were able to insert a gene at a precise site in the HSPCs with unprecedented efficiency rates ranging from 15 to 40 percent. That's truly a breakthrough.