Sci Adv: RISPR gene editing successfully treats cancer in live animals and is permanently effective

With the progress of science and technology and medical development, many diseases that used to make people helpless are now alleviated one by one, or even completely cured.
, however, cancer, as the "king of all diseases", is an incomprehensionable eddy that has not yet been completely defeated by mankind.
From traditional cancer treatments, such as radiotherapy and chemotherapy, to today's emerging cancer treatments , targeted drugs, lysovirus, and immunotherapy, advances in science offer new hope for cancer patients, but unfortunately these methods do not solve the cancer problem once and for all.
, it also forces humans to continue to develop newer, more efficient cancer treatments! On November 18, 2020, researchers at Tel Aviv University in Israel published a research paper in the journal Science Advances entitled CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy.
is the world's first to confirm that the CRISPR/Cas9 system is effective in treating metastasis cancer in live animals.
researchers have developed a new lipid nanoparticle (LNP)-based CIRSPR delivery system, CRISPR-LNP, and have shown good results in two types of invasive and incurable cancers, glioblastoma and ovarian cancer.
rapid development of CRISPR technology is changing the field of life science research and even the human world.
, CRISPR technology has been widely used in gene editing, gene therapy, nucleic acid positioning and nucleic acid detection and other fields.
October 7, 2020, two scientists who have made outstanding contributions to crispr gene editing, Emmanuele Charpentier and Jennifer A. Doudna, were awarded the 2020 Nobel Prize in Chemistry.
recent years, the rise of molecularly targeted drugs and immunotherapy has greatly improved cancer response, reducing drug toxicity and unresponsive response.
, however, the high recurrence rate and drug resistance of most types of cancer have prompted us to continue to develop new treatments.
it is worth noting that CRISPR-Cas9 gene editing techniques can in principle permanently destroy tumor survival genes, thus overcoming the repeated dose limits of traditional cancer therapies and improving the effectiveness of treatment.
, however, the use of CRISPR-Cas9 technology in cancer treatment has been hampered by inefficient tumor cell editing and the potential toxicity of existing delivery systems.
In this study, the team reported on the development of targeted non-viral lipid nanoparticles (LNP) delivery systems for therapeutic genome editing and evaluated them in two invasive and incurable cancer models, glioblastoma and ovarian cancer.
SPR-LNP design and construction co-author Professor Dan Peer said it was the first study in the world to demonstrate that CRISPR genome editing systems can effectively treat cancer in living animals, and it must be stressed that this is not chemotherapy, there are no side effects, and cancer cells treated in this way will never be active again.
cas9 molecular scissors cut off the cancer cell's DNA, removing it and permanently preventing the cancer cells from replicating.
to determine the feasibility of the technology in treating cancer, Professor Dan Peer and his team chose two of the deadliest cancers, glioblastoma and metastatic ovarian cancer.
glioblastoma is one of the most invasive brain cancers, with a life expectancy of 15 months and a five-year survival rate of only 3%.
researchers have shown that a single intra-brain injection treatment with CRISPR-LNP can double the life expectancy of mice with glioblastoma, inhibit tumor growth by 50%, and improve survival by 30%! CRISPR-LNP has good therapeutic effect on glioblastoma in mouse brains at the same time, ovarian cancer is an important cause of death in women and the deadliest cancer in the female reproductive system.
most patients were diagnosed with advanced ovarian cancer, by which time the cancer cells had spread throughout the body.
progress has been made in recent years, only a third of patients survive.
In order to treat diffuse ovarian cancer, CRISPR-LNP was also designed by the research team for antibody target delivery, celiac injection EGFR targeted CRISPR-LNP selectively ingested into the spreading ovarian tumor, the body can be up to 80% gene editing, inhibit tumor growth, and improve the total survival rate of mice 80%! The CRISPR-LNP design schematic CRISPR gene editing technology, which identifies and alters almost any gene fragment, has revolutionized our ability to destroy, repair, and even replace genes in a personalized manner, and although CRISPR has been widely used in research, clinical applications are still in their infancy because an effective delivery system is needed to safely and accurately deliver CRISPR to target cells.
CRISPR-LNP delivery system developed in this study can efficiently target tumor survival genes, an innovative therapy for treating malignant tumors that do not currently have an effective treatment.
It is worth mentioning that the application of CRISPR gene editing technology to cancer treatment is not the "one-man talk" of dan Peer's team, for example, on March 9, 2020, the Wuhan University Cheng Yu xue team was published in advanced Materials, an internationally renowned journal (A research paper entitled: Aptamer/Peptide-Functionalized-Editing System for Effective Immune Resaling Through Reversal of PD-L1-Mediated Cancer Cancer Pression was published.
The study developed a gene therapy delivery system based on natural polymers that can deliver CRISPR-Cas9 gene-edited granules specifically to the nuclei of tumor cells, then knock out β-catenin, reduce PD-L1 expression on tumor cells, reverse PD-L1-mediated tumor immune escape, and enhance T-cell lethality to tumors! All in all, CRISPR-LNP, a joint system based on CRISPR gene editing technology and lipid nanoparticles (LNP), demonstrated amazing potential in mouse models for the treatment of glioblastoma and metastatic ovarian cancer.
more importantly, the technology has broad application prospects and will open up numerous new possibilities for treating other types of cancer, rare genetic diseases and chronic viral diseases such as AIDS.
It is understood that Professor Dan Peer's team now plans to continue experiments on blood cancers and genetic diseases such as Duchia muscular dystrophy, and Professor Dan Peer is optimistic that the innovative treatment may take some time to be used in humans.
12 years ago, when we first talked about mRNA-based therapy, people thought it was science fiction.