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Fix-it kit for faulty genes

Yale Medicine Magazine, 2009 - Winter

Contents

School of Medicine researchers led by Peter M. Glazer, M.D. ’87, Ph.D. ’87, HS ’91, department chair and the Robert E. Hunter Professor of Therapeutic Radiology and professor of genetics, have found a new approach to gene therapy, opening up the possibility of new treatments for inherited hematologic diseases.

In the September 9 issue of the Proceedings of the National Academy of Sciences, the researchers report that they developed genetic “repair kits” consisting of chemically altered pieces of DNA, which bind to human genes and trigger the cell’s own repair systems to fix such mutated genes as the one that causes thalassemia, an inherited blood disease. The faulty gene was fixed even in human bone marrow cells, meaning that the genetic repair could be inherited by newly generated blood cells.

The new technique employs small pieces of synthetic DNA that are easy to insert into cells and do not require viruses for delivery.