Highlight: Efficient neuronal gene knockout using CRISPR

We recently wrote a research highlight related to the genome editing therapy pipeline. While therapy is an exciting use for genome editing, it is still in the early stages with only a few candidates progressing through clinical trials. In contrast, the use of technologies such as CRISPR in clinical research has been expanding rapidly. The use of CRISPR in research to understand biological processes and diseases will accelerate research and provide new therapies and diagnostics that might never ultimately be attributed to CRISPR.

A recent publication in Gene Therapy provides a very good example of this. In the article titled, “Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice,” the research team outlined a method that could change how we produce mouse models.

The paper documents how the research team were able to generate neuronal knockouts with very high efficiency compared to previous methods. This finding builds on a lot of work that has be done to edit the genome of non-dividing cells such as neurons which has been very challenging in the past.

As the authors rightly point out, the comparison between genetic tools available for prokaryotes and even invertebrates such as drosophila has historically vastly outnumbered those for vertebrate models. As such, the genetic and molecular understanding of these well studied model systems has advanced much faster than our understanding of invertebrates. CRISPR could be the technology to level the playing field and bring a whole new suite of advanced tools for studying complex invertebrate models.

The ability to efficiently create knockouts means that it will be much faster to generate new mouse models to study neurons in the future. This will help provide insight into everything from neuronal disease, cognitive processes as well as modelling emerging technologies on the human nervous system.

At Moligo, our mission is to enable advances in genome editing technology by providing industrial quantities of ultra-pure ssDNA oligos up to 10k bases. To find out if we can help you, please get in touch via the form below.

Reference

Hana, S., Peterson, M., McLaughlin, H. et al. Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice. Gene Ther (2021). https://doi.org/10.1038/s41434-021-00224-2