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Revolutionary CRISPR-based genome editing system treatment destroys cancer cells

Researchers at Tel Aviv University (TAU) have demonstrated that the CRISPR/Cas9 system is very effective in treating metastatic cancers, a significant step on the way to finding a cure for cancer. The researchers developed a novel lipid nanoparticle-based delivery system that specifically targets cancer cells and destroys them by genetic manipulation. The system, called CRISPR-LNPs, carries a genetic messenger (messenger RNA), which encodes for the CRISPR enzyme Cas9 that acts as molecular scissors that cut the cells' DNA.

The revolutionary work was conducted in the laboratory of Prof. Dan Peer, VP for R&D and Head of the Laboratory of Precision Nanomedicine at the Shmunis School of Biomedicine and Cancer Research at TAU. The research was conducted by Dr. Daniel Rosenblum together with Ph.D. student Anna Gutkin and colleagues at Prof. Peer's laboratory, in collaboration with Dr. Dinorah Friedmann-Morvinski from the School of Neurobiology, Biochemistry & Biophysics at TAU; Dr. Zvi R. Cohen, Director of the Neurosurgical Oncology Unit and Vice-Chair of the Department of Neurosurgery at the Sheba Medical Center; Dr. Mark A. Behlke, Chief Scientific Officer at IDT Inc. and his team; and Prof. Judy Lieberman of Boston Children's Hospital and Harvard Medical School.

The results of the groundbreaking study, which was funded by ICRF (Israel Cancer Research Fund), were published in November 2020 in Science Advances.

"This is the first study in the world to prove that the CRISPR genome editing system can be used to treat cancer effectively in a living animal," said Prof. Peer. "It must be emphasized that this is not chemotherapy. There are no side effects, and a cancer cell treated in this way will never become active again. The molecular scissors of Cas9 cut the cancer cell's DNA, thereby neutralizing it and permanently preventing replication."

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