Antisense oligonucleotides block flawed genetic instructions

Antisense oligonucleotides — also called antisense, oligos, or simply AONs— are pieces of genetic code that keep other genetic code from being processed. Designed to pair up with a particular sequence of DNA or RNA, AONs can change, block or destroy targeted genetic instructions in a variety of ways.

It didn’t happen during my childhood, when I walked around wobbly with a pediatric walker, smiling and conversing.

It didn’t happen in my sophomore year of high school, after an emergency room visit led to an 11-day hospital stay and a diagnosis of type 1 diabetes. It didn’t happen my senior year of high school, after a four-day hospital stay to implant a pacemaker to regulate my slow heartbeat.

It happened right after high school graduation, the summer before college began. I grew up.

MDA-supported research in Charcot-Marie-Tooth disease is focused on figuring out what goes wrong at the molecular level in CMT-affected axons or the myelin sheaths that surround them, rather than on attempting to fix the problem directly or preserving nerve function in spite of it. A central theme emerging from the last decade of research is that myelin and axons require constant signals from each other to stay functional.

The field of Charcot-Marie-Tooth disease (CMT) research is expanding, and people with the disease can help move it forward.

Discovery of the complicated genetics underlying CMT has made it clear that more studies are needed to correlate the progression, symptoms and outward manifestations of the disease with its specific genetic type.

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