Source: Massachusetts Institute of Technology
Summary: Researchers in a new study developed nanoparticles which can deliver the CRISPR genome-editing system and help in modifying genes specifically.
CRISPR (clustered regularly interspaced short palindromic repeats) is a genome editing system which contains a DNA-cutting enzyme called Cas9 and short RNA that helps in guiding the enzyme to a specific area in the genome to make its cut. Generally, researchers rely on viruses to carry the gene for Cas9 and also RNA guide strand. A nonviral high-pressure injection delivery systems were also developed but observed tissue damage at the target site. Researchers at Massachusetts Institute of Technology in a new study developed nanoparticles which can deliver the CRISPR genome-editing system and help in deleting disease-causing genes. The research findings were published in the journal Nature Biotechnology.
In this study, one of the genes targeted was Pcsk9 which regulates cholesterol levels. Mutations in the human version of this gene are linked to a rare disorder called dominant familial hypercholesterolemia. Recently two antibody drugs were approved by FDA to inhibit Pcsk9 but these drugs need to be taken for the rest of the patient’s life. Researchers by using the new nanoparticle delivery technique were able to delete out certain genes in about 80% percent of the liver cells and by far this is the best success rate achieved with CRISPR in adult animals. They are advancing this technique to use in human patients.
Assoc. Prof. Daniel Anderson said, “I think having a fully synthetic nanoparticle that can specifically turn genes off could be a powerful tool not just for Pcsk9 but for other diseases as well”,”The liver is a really important organ and also is a source of disease for many people. If you can reprogram the DNA of your liver while you’re still using it, we think there are many diseases that could be addressed.”
More Information: Hao Yin et al, Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing”, Nature Biotechnology (2017). nature.com/articles/doi:10.1038/nbt.4005