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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 sci-

ence 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-ING OUT DISEASE

CRISPR — formally known as CRISPR-

Cas9 — has been touted as an improve-

ment 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.

In April, scientists at the University of

Pennsylvania announced they had begun

using CRISPR for cancer treatments. The

first two patients — one with multiple

myeloma, the other with sarcoma —

had cells from their immune systems

removed. Researchers used CRISPR to

genetically edit the cells in the lab, and

then returned them back into their bodies.

Gene Therapy

Gets Clinical

BY LINDA MARSA

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