CRISPR-based therapy shows early promise for cancer

November 23, 2020 – Researchers say they used CRISPR, a new technology that allows scientists to modify a cell’s DNA, to destroy cancer cells in mice.

The first research conducted on two types of metastatic cancer – ovarian cancer and brain cancer – have not been tested in humans.

It suggests that lipid nanoparticles may be able to slip into cancer cells to provide instructions for the production of CRISPR enzymes that can break down the genetic material of those cells and kill them.

There has been a lot of excitement about the potential of CRISPR. But new treatments using technology have faced obstacles. It turns out that cells have a defense mechanism that kicks in to prevent their genetic material from being tampered with.

Researchers at Tel Aviv University, New York University, Harvard and a company called Integrated DNA Technologies have developed tiny lipid particles – about 50,000 times smaller than the width of a human hair – to provide the instructions for making the CRISPR-Cas9 enzyme in cells. Once inside, the cell uses the instructions to make the enzyme, which, in turn, would cut through the DNA of the cancer cells and kill them.

“We want to make sure this payload is concentrated in the right cell,” said Dan Peer, PhD, director of the Precision Nanomedicine Laboratory at Tel Aviv University in Israel. Peer and some of his co-authors revealed that they own patents on the technology and have a financial interest in developing the treatment.

“You don’t want to change nearby healthy cells. You want to change only the disease cells. What we call off target effects are potentially very high. What we have shown, with our strategy, if you look at the neighboring cells, we have found that there is no activity. There are no changes, ”says Peer, who also developed some of the technologies used in Pfizer’s COVID-19 vaccine.

In mice, Peer and his coauthors found that a single blow could slow tumor growth and improve survival in mice with brain and ovarian tumors that had spread.

He says he’d like to test the technology in people.

“We want to incorporate this into clinical trials,” he said.

The study was recently published in the journal Advances in science.

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Dan Peer, PhD, director, Laboratory of Precision NanoMedicine; vice president, research and development, Tel Aviv University, Israel.

Advances in science: “CRISPR-Cas9 Genome Editing Using Targeted Lipid Nanoparticles for Cancer Therapy.”

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