2020 Was the Turning Point for CRISPR

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...