NEW YORK (Reuters Health) - Highly specific engineered zinc-finger nucleases, designed to bind to sequences close to an X-linked SCID-causing mutation in the human IL2R gamma gene, resulted in a number of the cells undergoing gene correction, researchers report in an April 3rd online publication of Nature Genetics.
Senior investigator, Dr. Michael C. Holmes told Reuters Health that “by means of this technology we have advanced the field of targeted homologous recombination to levels of efficiency and specificity that potentially could make therapeutic applications feasible.”
Dr. Holmes of Sangamo BioSciences, Inc., Richmond, California and colleagues, note that the approach allows replacement of DNA segments with new copies.
The researchers were able to use the zinc-finger nucleases without selection to achieve a more than 18% yield of gene-modified human cells. “Remarkably,” the investigators write, about 7% of the cells acquired the desired genetic modification on both X chromosomes, with cell genotype accurately reflected at the messenger RNA and protein levels.”
The results “establish a general method for the rapid and permanent modification of the human genome at a specific location both in transformed and in primary cells,” the investigators conclude.
Source: Nat Genet 2005. [ Google search on this article ]
MeSH Headings: Biological Therapy : Genetic Engineering : Genetic Techniques : Investigative Techniques : Therapeutics : Gene Therapy : Zinc Fingers : Protein Structure, Secondary : Amino Acid Motifs : Analytical, Diagnostic and Therapeutic Techniques and Equipment Copyright © 2002 Reuters Limited. All rights reserved. Republication or redistribution of Reuters content, including by framing or similar means, is expressly prohibited without the prior written consent of Reuters. Reuters shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon. Reuters and the Reuters sphere logo are registered trademarks and trademarks of the Reuters group of companies around the world.
