CRISPR Gene-Editing Cancer Breakthrough in Human Trials

The CRISPR gene-editing tool for cancer will be used in its first-ever human trials by Chinese scientists in what they hope will lead to a breakthrough in developing improved therapies against the disease, Bloomberg reported.

Chinese researchers hope to use CRISPR -- short for "clustered regularly interspaced short palindromic repeats" -- to activate immune cells and introduce them back into bodies of patients to fight lung cancer, said Bloomberg. The scientists decided to treat the first group of three patients one at a time because of the potential risk of the new treatment.

CRISPR is a bacteria genome pattern that is part of its immune system. A set of enzymes called Cas (CRISPR-associated proteins) can precisely snip DNA and cut out invading viruses, noted tech website Gizmodo.

Chinese oncologist Lu You, of Sichuan University West China Hospital, is leading the group of scientists conducting the human trial.

The Chinese effort comes on the heels of a National Institutes of Health advisory committee in June approving the use of the technology known as CRISPR-Cas9 to augment cancer treatment that focuses on the patient's T cells, a type of immune cell, noted Nature.

"Cell therapies (for cancer) are so promising but the majority of people who get these therapies have a disease that relapses," Edward Stadtmauer, a physician at the University of Pennsylvania and study leader, told Nature.

Stadtmauer said gene editing could lead to treatment improvement along with eliminating some of its vulnerabilities to cancer and the body’s immune system. Nature said the University of Pennsylvania will manufacture the edited cells, and will recruit and treat patients alongside centers in California and Texas.

"As this point, you can start connecting the dots: Cas9 is an enzyme that snips DNA, and CRISPR is a collection of DNA sequences that tells Cas9 exactly where to snip," said Gizmodo writer Sarah Zhang. "All biologists have to do is feed Cas9 the right sequence, called a guide RNA, and boom, you can cut and paste bits of DNA sequence into the genome wherever you want."

Jason Moffat, a research chair in functional genomics of cancer at the University of Toronto, told CBC News that it appears that researchers are "pushing the technology really hard."

"It gives you the tools to ask questions about what things are doing that we could never do before," said Moffat. "It's really exciting."

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