A new study from scientists at the Institute of Bioorganic Chemistry in Poland has revealed an improved CRISPR/Cas9 approach when attacking Huntington's disease. The work was published today in Frontiers in Neuroscience.
"In our study, we further improve the CRISPR/Cas9 approach by using a nickase version of Cas9," says Marta Olejniczak, group leader of the study. "Because Cas9 nickases are known to be safe and specific, our approach provides an attractive treatment tool for Huntington's disease."
To check the safety and effectiveness of the treatment, Olejniczak's group tested a new variant of the Cas9 protein component in cellular models from a Huntington's patient. This new version of Cas9 acts as a nickase, an enzyme that cuts just one DNA strand instead of two, which increases the precision with which Cas9 can edit specific sequences of DNA.
"We demonstrated that excision of the repeat tract with the use of a Cas9 nickase pair resulted in inactivation of the huntingtin gene and abrogation of toxic protein synthesis in cellular models of Huntington's disease," says Olejniczak. "Our strategy is safe and efficient, and no sequence-specific side effects were observed."
This research is a step in the right direction in finding a possible treatment for Huntington's disease.