Photo by National Cancer Institute/ Unsplash
In a groundbreaking study, researchers from Tel Aviv University (TAU) have successfully used CRISPR gene-editing technology to eliminate 50% of head and neck cancer tumors in model animals. By precisely cutting a single gene from cancer cells, the team demonstrated a revolutionary approach to targeted cancer treatment.
The study was led by Dr. Razan Masarwy, MD, Ph.D., from the lab of Prof. Dan Peer, a world leader in mRNA-based drugs and nanomedicine. Prof. Peer is the Director of the Laboratory of Precision Nanomedicine, VP for Research and Development, and a member of the Shmunis School of Biomedicine and Cancer Research at TAU.
Published in the prestigious journal Advanced Science, these findings highlight the potential of CRISPR-based therapies to transform cancer treatment, paving the way for more effective, gene-targeted solutions in oncology.
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This breakthrough positions Tel Aviv University at the forefront of cancer research, offering new hope for future personalized cancer treatments. “Head and neck cancers are very common, ranking fifth in cancer mortality,” says Prof. Peer. “These are localized cancers, typically starting in the tongue, throat, or neck, which can later metastasize. If detected early, localized treatment can effectively target the tumor. Our aim was to use genetic editing of a single gene expressed in this type of cancer to collapse the entire pyramid of the cancerous cell. This gene is the cancer-specific SOX2, also expressed in other types of cancer, and overexpressed in these particular tumors.”
Prof. Peer and his colleagues are global pioneers in developing mRNA-based drugs encased in synthetic lipid particles that mimic biological membranes. In this study, the researchers synthesized special lipids that encapsulate the delivered CRISPR system in an RNA format. An antibody targeting a receptor against a protein named EGF was attached to the surface of these particles.
“These tumors are highly targeted,” explains Prof. Peer. “We targeted EGF because the cancer cells express the EGF receptor. Using our nano-lipid delivery system, we injected the drug directly into the tumor in a tumor model and successfully took out the gene – literally cutting it out from the cancer cell’s DNA with the CRISPR ‘scissors’. We were happy to observe the domino effect we had predicted. Following three injections spaced one week apart, 50% of the cancerous tumors simply disappeared after 84 days – which did not happen in the control group.”
In 2020, Prof. Peer and his team were the first in the world to use CRISPR to cut genes from cancer cells in mice and in a cell specific manner, and this is the first time they have applied it to head and neck cancers.
“Generally, CRISPR isn’t used for cancer because the assumption is that knocking out one gene wouldn’t collapse the whole pyramid. In this study we demonstrated that there are in fact some genes without which a cancer cell cannot survive, making them excellent targets for CRISPR therapy. Since cancer cells sometimes compensate with other genes, it’s possible that additional genes need to be cut out, or perhaps not. Theoretically, this approach could be effective against many types of cancer cells, and we are already working on additional cancer types, including myeloma, lymphoma, and liver cancer.”
