This week, for the first time, a hospital edited the genes of a patient, to cure him, one hopes. Forty-four-year-old Brian Madeux of Phoenix got an infusion of gene-editing liquid at UCSF Benioff Children's Hospital Oakland this week, as reported by AP. Brian has a genetic disease that's preventing him from making a needed enzyme. His disease costs $100,000 to $400,000 a year to treat, and the treatment doesn't prevent many of the worst symptoms. He's had 26 surgeries to help him manage his condition, but this treatment could cure him forever.

It's is the first time gene therapy has been used outside of the lab in an adult patient in a professional medical setting

CEO Sandy Macrae of Sangamo, the company responsible for the therapy, told AP, "We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending. It becomes part of your DNA and is there for the rest of your life."

Editing genes is going mainstream

"This is the first time in human history that we're no longer stuck with the genes we had at birth," says outspoken biohacker Dr. Josiah Zayner. He has modified his own genes numerous times. He makes his living selling do-it-yourself home biohacking kits. He has livestreamed stunts like injecting his own arm with gene-editing serums designed to modify his DNA so his muscles grow.

While some find Zayner an extreme example, he recently shared a pragmatic perspective with Rowan Jacobsen of Outside: "Let's imagine you're the worst person in the world and you want to hurt people with biologicals. First, you have to have the knowledge. Then, you have to have the facility. Then, you have to think about how it's going to spread. It would be an astounding feat. Could you kill one or two people? Sure. But you can do that with a f***ing kitchen knife." The article was called "Ultimate Life Hack." Indeed.

Gene editing next door

Whether you think Zayner is a prophet or a crackpot, the point is that you should get ready for gene editing and gene therapy coming to a hospital near you. The startups, scientists, and technologists behind these therapies are moving fast. It was only 2003 when human DNA was mapped for the first time. Less than 20 years later, a host of companies are bringing innovations to market. They make the old TV series The Incredible Hulk look like potential headline news.

What Zayner does as a Facebook stunt to create awareness, scientists are doing in private labs to create profits and provocative treatments. For example, this year, the National Institutes of Health approved gene editing in human embryos when it will prevent a serious disease or condition. A couple of months later, as reported by CNN, human embryos were gene edited to cure a genetic blood disease.

Some of the gene-editing innovations to watch include:

1.     GlaxoSmithKline's Strimvelis, which provides a cure for a fatal immune deficiency, is a treatment now approved in Europe. 

2.     Novartis drug Kymriah was just approved in the U.S. to edit cancer-fighting genes in children with leukemia.

3.    Technology by Cellectis allows cellular engineering, and a human trial of the company's work in editing blood cells will be presented at the upcoming American Society of Hematology. 

4.    Intellia Therapeutics has a significant pipeline of gene therapies that explore knocking out "bad genes" and often replacing them with "good genes." 

5.     Crispr Therapeutics, which operates in Cambridge, Massachusetts, Zug, and London, says its mission is "to cure diseases at the molecular level." Partners include deep pockets such as Bayer AG.

6.     Caribou Biosciences, which partners with Novartis and Intellia, has a technology that "enables simple, flexible targeting of any site in a genome." That means the company's up for creating anything, including "plants with enhanced traits," according to Caribou's website, as well as animal and human editing. 

7.     AveXis (AVXS) is a gene-therapy company in Bannockburn, Illinois, that went public last year. It's aggressively focused on curing spinal muscular atrophy.

8.    Editas, founded in 2013 in Cambridge, Massachusetts, is a visionary gene-therapy company with a pipeline of therapies for cancer and lung, liver, muscle, eye, and blood diseases. Editas's website gives you a sense of the mission and zeal it brings to its work: "There are few times in our lives when science astonishes us -- when we suddenly may be able to do something that once seemed out of reach. This is one of those moments."

9.  Audentes Therapeutics is focused on rare genetic diseases that affect muscles, including the heart, or reduce their victims to muscular weakness.

Customizing your DNA

Now that the barrier of editing genes in the hospital setting has been broken, these companies and more have pipelines of gene-editing innovation coming to a hospital near you in the next few years, as clinical trials are approved and new ways to cure formerly incurable diseases go mainstream in our lifetimes. The question is not if but when gene editing will be an option to manage a condition at the cellular level. Consider diabetes, which is a failure to produce insulin, or cancer, or sickle cell anemia.

Learn more about gene editing

No matter your position on gene editing, you owe it to yourself to get educated about the changes coming to medicine in the next few years. If you aren't sure regulation is keeping up, let your representatives know. If you'd like to see the science in action, many cities have open-door community biohacking labs you can check out. Printing chromosomes today isn't much harder than 3-D printing was a few years ago. Maybe you could even dream a little about what it means to free humanity from the DNA-as-destiny reality we've always known.

Published on: Nov 17, 2017
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