"They used synthetic proteins called zinc finger nucleases (ZFNs) to target a corrective gene at a specifically defined location in the genome of the X-CGD iPS cells. The iPS cells were then carefully screened to identify those containing a single copy of the corrective gene properly inserted only at the safe site. The researchers observed that some of the gene-corrected iPS cells could differentiate into neutrophils that produced normal levels of hydrogen peroxide, effectively 'correcting' the disease."Taking defective blood stem cells, and patching their programming!
Tuesday, June 21, 2011
ASC and Gene Therapy
A fascinating article from Science Daily: