More efficient gene targeting improves prospects for gene therapy
18 March 2007
The Nobel prize-winning MRC scientist Sir Aaron Klug and a team from an American company, Sangamo BioSciences, have announced a new way to target genes that may revolutionise medical research and pave the way for new treatments. Their findings are published in PNAS.
The scientists have developed a more efficient way to target genes using synthetic proteins. Many biological processes are determined by switching genes on or off. Genes give instructions for making the proteins involved in these processes. This regulation of gene expression is controlled by regulatory proteins called transcription factors.
Sir Aaron Klug, of the Medical Research Council’s Laboratory of Molecular Biology in Cambridge, devised these synthetic transcription factors called zinc-finger nucleases based on a naturally occuring design. These zinc-fingered nucleases have the capacity to recognise specific sequences of DNA which makes them extremely good at targeting particular genes without affecting others.
Sir Aaron explains: “The beauty of zinc-finger nucleases lies in their simplicity. Where other methods are long, arduous and often messy, it is relatively easy to switch off genes using this method. The zinc-finger design allows us to target a single gene, while the nuclease disrupts the gene. The single step process is extremely quick and reliable and opens up exciting possibilities for research and gene therapy.”
These findings confirm that this method of gene targeting will present an efficient option for scientists to create “knock-out” animals for research. Animal trials are already under way to use the technique to knock out the receptor of HIV in T-cells of AIDS patients, so leading to a supply of non-infectable T-cells, which will combat HIV and the other infections which occur in AIDS patients. Clinical trials are also in progress for stimulating the growth of new arteries in patients suffering from obstruction of the blood vessels in the limbs.
This is a powerful technology for intervening in gene expression in health or disease and therapeutic applications are planned for other diseases by Sangamo BioSciences.
Phone: 0207 670 5139
press.office@headoffice.mrc.ac.uk
Notes to Editors
Ref: MRC/08/16
