The Milbrandt and DiAntonio labs had a paper published recently on gene therapy that blocks peripheral damage in mice. The paper, “Gene therapy targeting SARM1 blocks pathological axon degeneration in mice,” appears in a recent issue of the Journal of Experimental Medicine. Stefanie Geisler, M.D., an assistant professor of neurology at Washington University School of Medicine, is first author on the paper. Jeff Milbrandt, M.D., Ph.D., the James S. McDonnell Professor of Genetics and head of the Department of Genetics, and Aaron DiAntonio, M.D., Ph.D., the Alan A. and Edith L. Wolff Professor of Developmental Biology, are co-senior authors on the paper.
“With our viral gene therapy, we delivered a mutated form of SARM1 that is not only inactive itself but also blocks normal SARM1 proteins that have become activated in mice with nerve injuries,” Dr. Milbrandt said in a press release. “For a long time, viral gene therapy was a pipe dream, but there are now a number of ongoing clinical trials in other disorders that suggest we are on a promising track.”