CRISPR against Cancer

A revolutionary method based on CRISPR/Cas9 technology helped destroy cancer cells

Denis Gordeev, Naked Science

Researchers from Tel Aviv University have demonstrated that a technique based on the CRISPR/Cas9 gene editing technique can be extremely effective in the treatment of metastatic cancer. An article about this is published in Science Advances (Rosenblum et al., CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy).

The authors have created a new molecular delivery system based on lipid nanoparticles – CRISPR-LNP. This system targets cancer cells and destroys them by disrupting the structure of nucleic acids. CRISPR-LNP contains the so-called matrix RNA, which encodes the enzyme CRISPR-Cas9; it, in turn, acts as a "molecular scissors", cutting the DNA of mutated cells.

"This is the first study that proves that the CRISPR genome editing system can be used to effectively treat cancer in a living organism," says lead author of the study Dan Peer. "It should be emphasized that this is not chemotherapy. There are no side effects here, and cancer cells treated in this way will never become active again. Cas9 molecular scissors cut the DNA of cancer cells, thereby neutralizing them and permanently preventing replication."

CRISPR-LNP preparation scheme. An alcohol mixture of lipids, CRISPR-LNP, CRISPR-Cas9 and guide RNA (sgRNA) is mixed in a special mixer to produce lipid nanoparticles. A drawing from an article in Science Advances.

To explore the possibilities of their development, Pir and his colleagues chose two of the deadliest types of cancer: glioblastoma and metastatic ovarian cancer. Glioblastoma is the most aggressive type of brain cancer, with a life expectancy of 15 months after diagnosis, and a five–year survival rate of only 3%. The researchers demonstrated that a single application of CRISPR-LNP doubled the average life expectancy of mice with glioblastoma, increasing their overall survival by about 30%.

Ovarian cancer is the most deadly oncological disease of the female reproductive system. Most patients are diagnosed with an advanced stage of the disease, when metastases have already spread through the body. Despite the progress in treatment achieved in recent years, only a third of patients survive. The use of CRISPR-LNP for the therapy of model mice with metastatic ovarian cancer increased the overall survival of rodents by 80%.

"CRISPR genome editing technology, capable of identifying and altering any genetic segment, has revolutionized our ability to disrupt, repair or even replace genes," explains Peer. "Despite widespread use in research, clinical implementation is still in its infancy, because for safe and accurate delivery of CRISPR to its cells-targets need an effective delivery system. The delivery system we have developed targets the DNA responsible for the survival of cancer cells. This is an innovative method of treating aggressive forms of cancer."

Now the authors of the work plan to study whether CRISPR-LNP will be effective in the treatment of blood cancer, as well as some genetic diseases like Duchenne muscular dystrophy. In the future, they want to use the new technology to treat AIDS and other chronic viral and hereditary diseases.

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