A New Era for Genetics

by Guy McKhann, M.D.

January 12, 2018

This is a column from Dana's print publication, Brain in the News.

Happy 2018 everyone.

I would like to begin by acknowledging a colleague, editor Andrew Kahn, who has been involved with producing Brain in the News (BitN) from the very beginning. Andrew has selected articles, provided me with information and guidance, and made BitN possible. He is leaving the Dana Foundation to become involved with communications regarding athletics at the University of Michigan. Right now, he is focusing on basketball but hopes to soon be involved with his dream job covering Michigan football. I don’t know if he will be a source for tickets to sold out games; I haven’t tried yet.

To start 2018, I would like to call attention to a new approach on modifying genetic diseases, based on Amy Ellis Nutt’s article in the Washington Post titled, “Phenomenal trial results may lead to a treatment for Huntington’s Disease, experts say” (http://wapo.st/2DThFSb). In Huntington’s disease, an abnormal gene (defined by Nancy Wexler, a member of the Dana Alliance for Brain Initiatives, in 1993) makes an abnormal protein that is toxic to nerve cells. Normally, a gene directs the formation of RNA, which acts as a messenger to cells to make a specific protein. In this new study, a team of international investigators interrupted things at the gene-to-RNA step, thus preventing the abnormal message from getting to cells and preventing the buildup of abnormal, toxic protein. In other words: they shut off the abnormal gene. Christopher Ross, director of the Baltimore Huntington’s Disease Center at Johns Hopkins, has suggested that based on animal studies, shutting of the abnormal gene will not only stop further damage caused by toxic protein, but also allow some healing to take place. 

This is not the only study directed at shutting off the abnormal gene and its subsequent functions in other diseases. I am sure that I will be featuring other studies as the year progresses. In the meantime, I have some homework to do as I learn more about these new approaches to modifying gene functions. The important point is that it is now possible to interrupt abnormal gene function. When started early, this makes it possible to prevent the disease from having any clinical manifestations. When applied later, it can stop further progression and, as Dr. Ross suggests, allow for healing to take place. A new era!