Researchers from Tel Aviv University have shown that the CRISPR genome editing system is highly effective in treating metastatic cancers, an important step on the road to finding a cure for cancer.
In a paper published this week, researchers demonstrated a new delivery system based on lipid nanoparticles that specifically targets cancer cells, and co-author Professor Dan Peer said it is the first study in the world to show that CRISPR / Cas9 can be used to effectively treat cancer in a live animal.
“It must be emphasized that this is not chemotherapy. There are no side effects and a cancer cell treated in this way will never be activated again, ”said Peer, vice president of R&D and director of the Precision Nanomedicine Laboratory at the Shmunis School of Biomedicine and Cancer Research.
“CRISPR genome editing technology, capable of identifying and altering any genetic segment, has revolutionized our ability to alter, repair or even replace genes in a personalized way.”
Peer’s team, which includes researchers from an Iowa company, Integrated DNA Technologies, and Harvard Medical School, chose two of the deadliest cancers: glioblastoma and metastatic ovarian cancer to examine the viability of the system. Glioblastoma is the most aggressive type of brain cancer, with a life expectancy of 15 months after diagnosis and a 5-year survival rate of only 3%.
The researchers showed that a single CRISPR-LNP treatment doubled the average life expectancy of mice with glioblastoma tumors, improving their overall survival rate by approximately 30%.
Ovarian cancer is one of the leading causes of death among women and the deadliest cancer of the female reproductive system. Despite progress in recent years, only a third of patients survive this disease, but CRISPR-LNP treatment in mice with metastatic ovarian cancer increased the overall survival rate by a whopping 80%.
“Despite its extensive use in research, clinical implementation is still in its infancy because an effective delivery system is needed to deliver CRISPR safely and accurately to its target cells,” Peer told Tel Aviv University News. “The delivery system we developed targets the DNA responsible for the survival of cancer cells. This is an innovative treatment for aggressive cancers that currently have no effective treatments. “
The system, called CRISPR-LNP, carries a genetic messenger (messenger RNA), which encodes the CRISPR Cas9 enzyme that acts like molecular scissors that cut DNA from cells. “The Cas9 molecular scissors cut the cancer cell’s DNA, neutralizing it and permanently preventing replication,” Peer explained.
The groundbreaking study was funded by the Israel Cancer Research Fund and was published this week. in scientific advances.
The researchers note that by demonstrating its potential in treating two aggressive cancers, the technology opens up numerous new possibilities for treating other types of cancer, as well as rare genetic diseases and chronic viral diseases such as AIDS. They intend to continue with experiments treating Duchenne muscular dystrophy, for example.
“It will probably be some time before the new treatment can be used in humans, but we are optimistic. The whole landscape of molecular drugs using messenger RNA (genetic messengers) is flourishing; in fact, most of the COVID-19 vaccines currently in development are based on this principle, ”says Peer.
“When we first talked about mRNA treatments twelve years ago, people thought it was science fiction. . . We are already negotiating with international corporations and foundations, with the aim of bringing the benefits of gene editing to human patients ”.
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