Forget designer babies. Here’s how CRISPR is changing lives

Even then, patients won’t get the treatment if insurers and governments negotiate to pay for it. It is a real danger. For example, a specific gene therapy for beta-thalassemia developed by Bluebird Bio was withdrawn from the European market after governments there refused to pay the $1.8 million price tag.

CRISPR 2.0

The first CRISPR treatments were limited in another way. Most use the tool to damage DNA, essentially destroying genes—a process famously described by Harvard biologist George Church as “genome destruction.”

Treatments that try to break genes include those designed to try to destroy HIV. The other is what Gray found. By breaking a small piece of DNA, the treatment unlocks the second hemoglobin gene that people normally only use as children. Because hemoglobin is the wrong protein in the sickle cell, making another copy solves the problem.

According to Liu’s analysis, two-thirds of current research is aimed at “disabling” genes in this way.

Liu’s lab is working on next-generation gene editing approaches. These tools use the CRISPR protein, but are designed not to cut the DNA helix, but rather to systematically swap individual genetic letters or make large edits. These are known as “base editors”.

Luis Montoliu, a geneticist at Spain’s National Center for Biotechnology, said these new versions of CRISPR are “less dangerous and perform better,” although delivering them “to the right cell in the body” is difficult.

Motoliu is using base editors in his lab to treat mice with albinism, in some cases from birth. He said it’s a step toward making the treatment available to newborns, even if it doesn’t change their skin color. Instead, he dreams of injecting Leu molecules into their eyes to correct the severe vision problems that albinism can cause.

So far, however, the albinism project is not a business. And that points to one of the biggest limits to CRISPR’s impact now and in the future. Almost all of the CRISPR trials in progress are aimed at cancer or sickle cell disease, and several companies are pursuing similar problems.