Release date: 2017-11-16 The gene editing system CRISPR-Cas9 is exciting because of its potential use as a means of eliminating pathogenic variability in DNA. However, most CRISPR systems rely on viruses to integrate the Cas9 complex into cells, and then perform DNA sequence excision. Such viral vector systems are at risk: some patients already have antiviral antibodies in their bodies, or they can produce these antibodies to increase the likelihood of immune rejection. Scientists at the Massachusetts Institute of Technology have developed an alternative to the Cas9 viral vector: the nanoparticle method, which integrates CRISPR mechanisms into cells and edits their genome. Moreover, they demonstrated the hope of the system by targeting the PCSK9 gene, which leads to a rare high cholesterol genetic morphology. In mice, they used a nanoparticle-based CRISPR system to eliminate PCSK9 genes in more than 80% of hepatocytes. The study was published in the journal Nature Biotechnology. The MIT research team led by chemical engineering professor Daniel Anderson has previously developed a non-viral CRISPR vector system that requires high pressure injection into the liver, but this increases the risk of organ damage. Later they used a nanoparticle carrier system to eliminate the need for injection, but still needed to use a virus to deliver a component, an RNA induction called sgRNA. So they analyzed the link between sgRNA and Cas9 and tried chemical modification until they had a mixture that could be used instead of the virus without interfering with the interaction between the two molecules. When they injected nanoparticles into mice to cut PCSK9, they found that PCSK9 protein almost disappeared and cholesterol levels in animals decreased by 35%. There are two drugs on the market for PCSK9 in patients with familial hypercholesterolemia. This high cholesterol form usually does not respond to traditional statins: Repatha sold by Amgen and Praluent developed by Sanofi/Regeneration. But these drugs must be taken regularly. The MIT team believes their approach can be used to develop a one-time treatment for the disease. The MIT team is one of many research groups looking for ways to turn CRISPR into a usable therapy. To date, CRISPR-Cas9 has proven to be impractical in non-dividing cells such as the brain, so scientists at the Max Planck Institute of Neuroscience in Florida are working on a neuronal genetic editing system alternative. The use of CRISPR to edit RNA rather than DNA has been explored as a way to not only expand the potential use of the technology, but also to make the editing of genes reversible. The next step for the MIT research group will be to identify more liver diseases that may be targeted based on the nanoparticle CRISPR system and further develop its human testing technology. The research team included Robert Langer, a professor at the Massachusetts Institute of Technology, who founded several biotech startups inspired by the school's findings. Anderson said in a press release: "I think that having a synthetic nanoparticle that can specifically transform genes can not only be a powerful tool for PCSK9, but also a powerful tool for other diseases. The liver is a very important organ. It is also a source of disease for many people. If you reprogram DNA during the liver work, we think there are many diseases that can be solved." Source: Sina Pharmaceutical News GMP Certificated Immune Globulin Injection Supplier in China Hepatitis B Immunoglobulin,Hep B Immunoglobulin,Hepatitis B Immunoglobulin Vaccine,Hepatitis Immune Globulin FOSHAN PHARMA CO., LTD. , https://www.foshanmedicine.com