Nanoparticle Delivery Technology Crispr-Gold Helps Crispr Treatment of Autism

CRISPR is a repeating sequence in the prokaryotic genome. It is an immune weapon produced by the struggle between bacteria and viruses in the history of life evolution. Simply put, the virus can integrate its own genes into the bacterial genome and use the bacterial cell tools for its own the gene duplication service. In order to remove the foreign invading genes of the virus, Bacteria evolved the CRISPR-Cas9 system. With this system, bacteria can quietly remove the virus gene from its own chromosome, which is the unique immune system of bacteria.

Microbiologists have mastered the immune function of bacteria with a variety of excised foreign virus genes. The more typical model relies on a complex that can direct the target DNA sequence under the guidance of a stretch of RNA and then excise the sequence. Many bacterial immune complexes are relatively complex, in which scientists mastered the manipulation of a protein, Cas9, and subsequently removed a variety of target cell DNA. This technique is known as the CRISPR/Cas9 gene editing system. Because of its precision, low cost, and ease of use, the technology is quickly becoming the hottest technology in life sciences.

Through the research on CRISPR-Cas9 system, the potential of CRISPR technology in medical health depends on three DNA editing components – Cas9 DNA cleavage enzyme, guide RNA and DNA donor. The efficient delivery of these components to specific target cells is key to the functioning of the Cas9 system. In the past, viruses were used to transport these molecules, but there were safety risks, limited traffic, and reduced editing efficiency of CRISPR components.

Researchers at the University of California, Berkeley, invented a technology called CRISPR-Gold that uses gold nanoparticles to deliver DNA-cut Cas9 enzymes to the brain, enabling researchers to edit neurotransmitter receptor genes and reduce the exaggerated repetitive behavior of fragility X syndrome (FXS) occurs with this onset of symptoms. This study can be used as a pre-study for autism research because people with clinically autism spectrum disorders (ASD) often have problems that are difficult to interact with others and exhibit exaggerated repetitive behavior. ASD pathogeny often has multiple causes, including variations in multiple genes. To simplify the complexity of the disease, researchers can first explore the cause and potential treatments with a single-gene disease such as FXS. There are currently no clinical treatments for autism, and many clinical trials for small molecule treatments targeting proteins have failed. The study was the first to be able to edit autism-causing genes in the brain, and it greatly improved the behavior of mice in the experiment.

In this study, the researchers used the CRISPR-Gold technology developed by Murthy, which binds the Cas9 complex through a DNA-coated gold nanoparticle, including a gene-cleaving enzyme and a guide RNA. This combination is encapsulated in a polymer to help enter the proper cells. The researchers believe that this is called CRISPR-Gold technology has more advantages than other methods of implanting Cas9 into the body (such as using viral transmission). For example, previous researchers have been able to deliver the Cas9 gene to neurons by using adeno-associated virus (AAV), but because the gene continues to produce Cas9, excessive Cas9 enzymes cause other genes to be knocked out and cause Potential problems. In contrast, CRISPR-Gold itself carries the Cas9 complex (including the purified Cas9 protein and the guide RNA) directly into the cell, where it only undergoes several gene cuts and then disappears. Because scientists can control the doses that are desired to be injected, it is possible to minimize the side effects of CRISPR, such as off-target effects.

Further research has found that CRISPR-Gold can transport Cas9 to various types of cells in the muscles and brain. Therefore, this technology opens the door to clinical treatment of opioid addiction, neuropathic pain, schizophrenia and seizures.