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Researchers at Tel Aviv University have shown that the CRISPR genome editing system is highly effective in treating metastatic cancers, an important step in the search for a cure for cancer.
In a paper published this week, researchers demonstrated a novel nanoparticle-based delivery system that specifically targets cancer cells – and co-author Prof Dan Peer said this was the world’s first study to prove that CRISPR / Cas9 could be used. to effectively treat cancer in a living animal.
“It should be noted that this is not chemotherapy. There are no side effects and a cancer cell treated in this way will never become active again, ”said Peer, vice president of R&D and head of the Precision Nanomedicine Laboratory at the Shmunis School of Biomedicine and Cancer. Research.
“CRISPR genome editing technology, capable of identifying and modifying any genetic segment, has revolutionized our ability to disrupt, 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 feasibility of the system. 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 just 3%.
Researchers demonstrated that a single treatment with CRISPR-LNPs doubled the average life expectancy of mice with glioblastoma tumors, improving their overall survival rate by about 30%.
Ovarian cancer is a major cause of death in women and the deadliest cancer of the female reproductive system. Despite the progress made in recent years, only one-third of patients survive the disease, but CRISPR-LNP treatment in mice with metastatic ovarian cancer increased the overall survival rate by 80%.
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“Despite its extensive use in research, clinical implementation is still in its infancy as an efficient delivery system is required to safely and accurately deliver CRISPR to its target cells,” Peer told The News. Tel Aviv University. “The delivery system we have developed targets the DNA responsible for the survival of cancer cells. It is an innovative treatment for aggressive cancers that have no effective treatment today. “
The system, called CRISPR-LNP, carries a genetic messenger (messenger RNA), which encodes the CRISPR Cas9 enzyme which acts like molecular scissors that cut the DNA of cells. “Cas9’s molecular scissors cut the DNA of the cancer cell, thus neutralizing it and permanently preventing replication,” Peer explained.
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The groundbreaking study was funded by the Israel Cancer Research Fund and published this week in Science Advances.
The researchers note that by demonstrating its potential in treating two aggressive cancers, the technology opens up many new possibilities for treating other types of cancer, as well as rare genetic diseases and chronic viral diseases like AIDS. They intend to carry out experiments dealing with, for example, Duchenne muscular dystrophy.
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“It will probably be some time before the new treatment can be used in humans, but we are optimistic. The whole scene of molecular drugs that use messenger RNA (genetic messengers) is booming – 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 companies and foundations, with the aim of bringing the benefits of gene editing to human patients.
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