Welcome to the Gene Editing Revolution

Welcome to the Gene Editing Revolution

It sounds like science fiction: Snip out a bad gene, insert a good one, and stop cancer in its tracks. But this fall, a British baby named Layla Richards made history when doctors used genetic engineering technology to knock out the cancer that threatened her young life.

Layla had a severe and unusual form of acute lymphoblastic leukemia. Diagnosed at 14 weeks, the baby girl received chemotherapy and a bone marrow transplant. But her cancer was aggressive and resisted treatment. Layla’s parents asked doctors to try anything – and physicians at London’s Great Ormond Street Hospital turned to a technique never before used against cancer in humans: Gene editing.

The gene-editing technology, so new it had only been studied in mice, removed a gene from the spreading cancer cells that protected them against destruction by Layla’s immune system and it beefed up cancer-fighting immune cells in her system so they could seek out and destroy the now-vulnerable cancer cells. The beefed-up immune cells also had genes edited into them that were able to protect them from the drugs Layla was taking. Soon after her first birthday, Layla received the infusion.

For three months, the enhanced immune cells roamed her body, wiping out her cancerous cells. Layla then had a successful bone marrow transplant. Doctors announced in November that she was cancer-free, calling it "almost a miracle".

Gene editing is a fast-moving field that holds promise for improving health in many ways. A few weeks before Layla’s story made headlines, the Cleveland Clinic, where Dr. Mike is Chief Wellness Officer, named a gene-editing technique to the Top 10 Medical Innovations for 2016. The list is usually reserved for breakthroughs that doctors and patients can currently use. But gene editing has such amazing potential to change clinical care in 2016 that the Clinic’s physicians voted it a top-10 spot.

Back to the Lab
Someday soon gene editing could help wipe out illnesses that are caused by a single, inherited gene; diseases like cystic fibrosis, hemophilia or breast cancers triggered by one of the BRCA genes. And it may help to edit-out problem genes that develop later in life, called somatic mutations. It’s also exciting because the ability to edit genes lets researchers learn more than ever about problems made worse by multiple genes, such as heart disease, diabetes and obesity.

Scientists have several gene-editing tools at their disposal. Layla’s treatment used one called TALENs, another has the catchy name, zinc fingers. We think the future’s very bright for one called CRISPR. It uses a protein that knows how to locate specific genes, then edit or snip them out. It’s cheap (as little as $30), fast and precise. That’s important because the 23 pairs of chromosomes in the human genome contain 30,000 genes.

That is some of the good work that’s already underway. Here’s a short list of some exciting projects:

Obesity gene: Researchers at Harvard University are looking at whether CRISPR can be used to snip out an “obesity gene” that governs the metabolism of fat cells.

Pancreatic cancer: Stanford University researchers are using CRISPR to study how this quick-spreading, hard-to-treat cancer develops.

High blood pressure: Lifestyle choices play a big role in blood-pressure problems, your genes are involved, too. University of Iowa scientists are zeroing in on specific genes in hopes of learning how to edit out those that promote high blood pressure.

Heart disease: The Montreal Heart Institute is leading an international effort to pinpoint culprits and how they work. They can edit one gene -- or a family of genes -- to see if that helps to eliminate heart disease.

Alzheimer's disease: Some genes boost risk for early Alzheimer’s, while another -- a variant of the APOE4 gene carried by one in five of us -- doubles risk for this dementia later in life. Massachusetts General Hospital researchers are looking to gene editing to replace that APOE4 gene and find other ways to treat this form of dementia.

Medically reviewed in July 2019.

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