The CRISPR/Cas9 gene editing system has become a powerful research tool uncovering the function of hundreds of genes and is currently being explored as a therapeutic tool for the treatment of various diseases. However, some technical hurdles remain before it can be practical for clinical applications. CRISPR/Cas9 is a large molecular complex, containing both a nuclease (Cas9) that can cut through both strands of a targeted genomic sequence, and an engineered 'single-guide' RNA (sgRNA) that scans the genome to help the nuclease find that specific sequence to be edited. Since it is a large molecular complex, it is difficult to deliver CRISPR/Cas9 directly into the nucleus of the cell, where it can do its work. Others have packed the editing molecules into viruses, polymers, and different types of nanoparticles to get them into the nucleus, but the low efficiency of tranfer has limited their use and potency for clinical applications.
The lipid nanoparticles described in the study encapsulate messenger RNA (mRNA) encoding Cas9. Once the contents of the nanoparticles -- including the sgRNA -- are released into the cell. The cell's protein-making machinery takes over and creates Cas9 from the mRNA template, completing the gene editing kit. A unique feature of the nanoparticles is made of synthetic lipids comprising disulfide bonds in the fatty chain. When the particles enter the cell, the environment within the cell breaks open the disulfide bond to disassemble the nanoparticles and the contents are quickly and efficiently released into the cell.
Trials - CRISPR - Therapies - Qiaobing - Xu
"We are just starting to see human clinical trials for CRISPR therapies," said Qiaobing Xu, co-corresponding author of the study and associate professor of biomedical engineering at Tufts University....
Wake Up To Breaking News!