In a paper published this week, the researchers demonstrated a novel lipid nanoparticle-based delivery system that specifically targets cancer cells—and co-author Prof. Dan Peer said it's the first study in the world to prove that the CRISPR/Cas9 can be used to treat cancer effectively in a living 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 become active again," said Peer, the VP for R&D and Head of the Laboratory of Precision Nanomedicine at the Shmunis School of Biomedicine and Cancer Research.
"The CRISPR genome editing technology, capable of identifying and altering any genetic segment, has revolutionized our ability to disrupt, repair or even replace genes in a personalized manner."
Peer's team that 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 system's feasibility. 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 treatment with CRISPR-LNPs doubled the average life expectancy of mice with glioblastoma tumors, improving their overall survival rate by about 30%.