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Gene Therapies Make it to Clinical Trials

After years of ethical debates and breakthroughs in the lab, CRISPR has finally made its way to clinical trials. Researchers are now looking at whether the DNA-editing tool, as well as more conventional gene therapies, can effectively treat a wide array of heritable disorders and even cancers.

“There’s been a convergence of the science getting better, the manufacturing getting much better, and money being available for these kinds of studies,” says Cynthia Dunbar, a senior investigator at the National Heart, Lung, and Blood Institute. “It’s truly come of age.”

CRISPR — formally known as CRISPR-Cas9 — has been touted as an improvement over conventional gene therapy because of its potential precision. CRISPR (clustered regularly interspaced short palindromic repeats) is a genetic code that, contained in a strand of RNA and paired with the enzyme Cas9, acts like molecular scissors that can target and snip out specific genes. Add a template for a healthy gene, and CRISPR’s cut can allow the cell to replace a defective gene with a healthy one.

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