Sticking It To Rogue DNA
Michigan Technological University chemistry professor Shiyue Fang and his team have developed a process that could lead to stickier—and better—gene therapy drugs.
The drugs, called antisense DNA, are made from short, single strands of synthetic DNA. They work by blocking cells from making harmful proteins, which can cause maladies ranging from cancer to Ebola to HIV-AIDS. Only a couple of these synthetic DNA drugs are on the market, but a number are in clinical trials, including a potential treatment for ALS, also known as Lou Gehrig’s disease.
Organisms can inject harmful proteins into our bodies, and so can mutations in our own genetic material.
Here’s how it works in a nutshell. When all goes well, messenger RNA molecules in our cells produce the good proteins that are essential to life. However, when mutations occur, messenger RNA can go rogue and start making proteins that make us sick.
Drugs made from synthetic DNA are tailored to grab onto these mutant messenger RNA molecules, binding to them and preventing them from churning out toxic proteins. However, a serious shortfall with synthetic DNA is that it can be wimpy. Sometimes it loosens its grip, setting the messenger RNA free to resume its dirty work.
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