2020 Was the Turning Point for CRISPR

Scientists took huge strides toward using the gene-editing tool for medical treatments

Photo: Yuichiro Chino/Getty Images

Amid a raging global pandemic, the field of gene editing made major strides in 2020. For years, scientists have been breathlessly hopeful about the potential of the gene-editing tool CRISPR to transform medicine. In 2020, some of CRISPR’s first real achievements finally came to light — and two of CRISPR’s inventors won the Nobel Prize.

The idea behind CRISPR-based medicine sounds simple: By tweaking a disease-causing gene, a disease could be treated at its source — and possibly even cured. The other allure of gene editing for medical reasons is its permanence. Instead of a lifetime of drugs, patients with rare and chronic diseases like muscular dystrophy or cystic fibrosis could instead get a one-time treatment that could have benefits for life.

This idea has proven difficult to realize. For one, scientists have to figure out how to get the gene-editing molecules to the right cells in the body. Once there, the molecules need to modify enough cells in order to have an impact on the disease. Both of these things need to happen without causing unpleasant or toxic side effects that would make the treatment too risky. With advances in CRISPR technology, scientists showed this year that we’re getting closer to gene-editing cures.

“2020 is the year that we have definitive proof that we are headed to a future where we as a species will genetically engineer human beings for purposes of treating disease or preventing them from developing disease,” Fyodor Urnov, PhD, a gene-editing expert and professor of molecular and cell biology at the University of California, Berkeley, tells Future Human.

Here’s why 2020 was such a milestone year for CRISPR.

CRISPR has eliminated symptoms of genetic blood diseases in patients

In July 2019, scientists at Vertex Pharmaceuticals of Boston and CRISPR Therapeutics of Cambridge, Massachusetts used a groundbreaking approach to treat a woman with sickle cell disease, an inherited blood disorder that affects 100,000 Americans — most of them Black — and often leads to early death.